[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"bleeding-disorder\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:bleeding-disorder":24},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,13,0,[8,49,81,111,140,179,212,236,246,270,297,325,357],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":17,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":21,"conditions":22,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":38,"startDateStruct":41,"completionDateStruct":43,"leadSponsor":45,"locationsCount":48},"100054158","athn-transcends-a-natural-history-study-of-non-neoplastic-hematologic-disorders-100054158",false,"NCT04398628","ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders","ATHN Transcends: A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment in People With Non-Neoplastic Hematologic Disorders","Participants who meet the following inclusion criteria and none of the exclusion criteria are eligible for enrollment in one of the open disease-specific arms.\n\nInclusion Criteria:\n\n1. Any age\n2. Having a congenital or acquired blood disorder; or\n3. Having a bleeding phenotype as indicated by an age adjusted abnormal ISTH Bleeding Assessment Tool score with an unknown diagnosis; or\n4. Connective tissue disorder with bleeding tendency as indicated by an age adjusted abnormal ISTH Bleeding Assessment Tool score.\n5. Eligible for a currently active disease-specific arm.\n6. Concurrent enrollment in the ATHNdataset or current ATHNdataset participant.\n\nExclusion Criteria:\n\n1\\. Does not qualify for inclusion in a currently activedisease-specific arm; participants may be eligible to enroll as future cohorts and arms are activated; 2. Unable to give informed consent or assent 3. Unwilling to perform study procedures\n\nCohort Participant Selection\n\nEach participant is to be enrolled in the cohort for which they qualify as defined below.\n\nHemophilia Cohort\n\nInclusion Criteria:\n\nParticipants who meet any of the following inclusion criteria are eligible for enrollment into this cohort:\n\n1. Factor VIII or factor IX activity \\\u003C50%, without another explanation for low clotting factor other than congenital hemophilia or being a known carrier for congenital hemophilia; OR\n2. Carrier for congenital hemophilia with a factor VIII \\>=50% or factor IX activity \\>=50% with or without a bleeding phenotype as indicated by an ISTH Bleeding Assessment Tool score of ≥4 for adult males, ≥6 for adult females, or ≥3 for children younger than 18 years OR\n3. Known congenital hemophilia that have a factor level \\>50% after receiving vector, OR 4. Acquired hemophilia.\n\nExclusion Criteria:\n\nNone\n\nVon Willebrand Disease Cohort\n\nInclusion Criteria:\n\nParticipants who meet the following inclusion criteria are eligible for enrollment into this cohort:\n\n1\\. Meeting the definition of VWD or low VWF per most recent international guidelines\n\nExclusion Criteria:\n\nNone\n\nCongenital Platelet Disorders Cohort\n\nInclusion Criteria:\n\nParticipants who meet the following inclusion criteria are eligible for enrollment into this cohort:\n\n1. Abnormalities of platelet function a. Glanzmann thrombasthenia (GPIIb or GPIIIa) b. Bernard-Soulier syndrome (GPIbalpha, GPIbbeta, or GPIX)\n2. Abnormalities of platelet granules\n3. Abnormalities of platelet signal transduction\n4. Abnormalities of platelet secretion\n5. Collagen Receptor Defect\n6. ADP Receptor Defect\n7. Thromboxane Receptor Defect\n8. Giant Platelet Disorder\n9. Abnormalities in platelet aggregation testing due to another or unknown cause (not drug related)\n\nExclusion Criteria:\n\n1\\. Platelet disorders secondary to medications or other substances\n\nRare Disorders Cohort\n\nInclusion Criteria:\n\nParticipants who meet the following inclusion criteria are eligible for enrollment into this cohort:\n\n1\\. Have an established Rare Coagulation Disorder (RCD) diagnosis of one of the following:\n\n1. PAI-1 deficiency\n2. Factor I, II, V, VII, X, XI, XIII deficiencies\n3. Combined FV and FVIII deficiency\n4. Plasminogen deficiency\n5. Decreased tissue plasminogen activator\n6. Afibrinogenemia\u002Fhypofibrinogenemia\u002Fdysfibrinogenemia\n7. Thrombotic Thrombocytopenia Purpura or Congenital Hemolytic Uremic Syndrome\n8. Wiskott-Aldrich\n9. Methylenetetrahydrofolate Reductase Deficiency\n\nExclusion Criteria:\n\nNone\n\nBleeding NOS Cohort\n\nInclusion Criteria:\n\nParticipants who meet the following inclusion criteria are eligible for enrollment into this cohort:\n\n1. Have a bleeding phenotype as indicated by an ISTH Bleeding Assessment Tool score of ≥4 for adult males, ≥6 for adult females, or ≥3 for children younger than 18 years with an unknown diagnosis, OR\n2. Connective tissue disorder with bleeding tendency as indicated by an ISTH Bleeding Assessment Tool score of ≥4 for adult males, ≥6 for adult females, or ≥3 for children younger than 18 years.\n\nExclusion Criteria:\n\nNone\n\nThrombosis\u002FThrombophilia Cohort\n\nInclusion Criteria\n\nParticipants who meet the following inclusion criteria are eligible for enrollment into this cohort:\n\n1\\. Have a prior history of arterial or venous thrombosis. 2. Participants with a known congenital or acquired thrombophilia with or without thrombosis.\n\na. Common congenital thrombophilias: i. Protein C deficiency ii. Protein S deficiency iii. Antithrombin deficiency iv. Factor V Leiden v. Prothrombin gene mutation b. Rare genetic factors i. Hyperhomocysteinemia c. Indeterminate genetic factors i. Elevated factor VIII ii. Elevated factor IX iii. Elevated factor XI iv. Elevated lipoprotein (a) d. Acquired thrombophilias i. Lupus anticoagulant ii. Anti-cardiolipin antibodies\u002FBeta2 glycoprotein antibodies iii. Antiphospholipid syndrome\n\nExclusion Criteria Acquired thrombophilia secondary to medications (birth control pills or hormone replacement therapy), overweight or obesity, smoking, cancer, pregnancy, surgery, injury, prolonged inactivity\u002Fbedrest, heart failure, inflammatory bowel disease, or kidney disease\n\nNon-Neoplastic Hematologic Conditions Cohort\n\nInclusion Criteria\n\nParticipants who meet the following inclusion criteria are eligible for enrollment into this cohort:\n\n1\\. Having any congenital or acquired non-neoplastic hematologic disorder not included in any other cohort\n\nExclusion Criteria None\n\nArm\u002FModule Participant Selection\n\nPreviously Untreated Patients Arm\n\nInclusion Criteria:\n\n1. Diagnosis of congenital hemophilia A (FVIII \\\u003C40%) or hemophilia B (FIX \\\u003C40% or below lower limit for age)\n2. Age \\\u003C18 years at time of enrollment\n3. Parent or authorized guardian or legally authorized representative (LAR) can provide informed consent\n4. Care established at one of the ATHN Transcends participating HTCs\n5. Clotting Factor Concentrate (CFC) exposure, fresh frozen plasma (FFP), cryoprecipitate, and single donor platelets \\\u003C3 exposure days (ED)\n\nExclusion Criteria\n\n1. Concomitant diagnosis with another bleeding disorder\n2. History of a confirmed, positive inhibitor\n\nINHIBIT Module\n\nInclusion Criteria:\n\n1\\. Diagnosis of severe factor VIII deficiency with baseline factor VIII level \\\u003C1% 2. Initiating or plan to initiate prophylaxis with emicizumab or factor replacement 3. Factor concentrate exposure, Fresh Frozen Plasma (FFP), cryoprecipitate, and single donor platelets ≤3 EDs 4. ≤5 years of age\n\nExclusion Criteria\n\n1. Concomitant diagnosis with bleeding disorder other than hemophilia A\n2. Immune disorder\n3. Previous history or presence of factor VIII inhibitor. A confirmed, positive inhibitor is defined as two consecutive positive inhibitor titers (≥ 0.6 BU) that result in changes in treatment recommendations.\n\nEfanesoctocog alfa (ALTUVIIIO®) Module\n\nInclusion criteria:\n\n1. Ability of the potential participant's legally authorized representative (e.g., their parent or legal guardian) to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use confidential health information in accordance with national and local participant privacy regulation.\n2. People with severe HA with a baseline FVIII activity of less than 1%. (While inclusion for participation in ATHN Transcends lists \\\u003C5% FVIII activity, this proposed module will limit enrollment to people with FVIII activity levels of \\\u003C1%.) Other severities may be included per ATHN Transcends PI approval.\n3. \\\u003C18 years of age.\n4. No history of a confirmed, positive FVIII inhibitor.\n5. Sex assigned at birth of male, female, or intersex.\n6. Participants should have no more than three (3) exposure days of blood products (fresh frozen plasma, cryoprecipitate, or platelets), no more than three (3) doses of any FVIII concentrate other than efanesoctocog alfa, and up to three (3) doses of efanesoctocog alfa prior to enrollment.\n7. Site PI confirmed all inclusion criteria has been met.\n\nExclusion criteria:\n\n1. Not meeting all the inclusion criteria; confirmed by site PI.\n2. Any exposure to blood products or FVIII replacement products except as described in the inclusion criteria.\n3. History of positive inhibitor testing.\n4. History of hypersensitivity reactions associated with efanesoctocog alfa administration.\n5. Other coagulation disorder(s) in addition to Hemophilia A.\n6. Any concurrent clinically significant major disease such as cancer that, in the opinion of the investigator, would make the participant unsuitable for enrollment.\n7. Concurrent systemic treatment with chemotherapy and\u002For other immunosuppressant medications. Use of corticosteroids for the treatment of asthma or management of acute allergic or otherwise life-threatening episodes is allowed except for systemic corticosteroid treatment given to children daily or on an alternate day schedule at \\> 2 mg\u002Fkg\u002Fday of prednisone or its equivalent or \\> 20 mg\u002Fday if the duration is longer than 14 days.\n8. Enrollment in a concurrent clinical interventional drug study.\n9. Intake of an Investigational Medicinal Product within three (3) months prior to inclusion in this study.\n10. Inability to comply with study requirements.\n11. Other, unspecified reasons that, in the investigator's opinion, make the participant unsuitable for enrollment.\n\nHemophilia Natural History Arm\n\nInclusion Criteria\n\n1. Congenital or acquired hemophilia A or B of any severity with or without inhibitors receiving a current therapy, a non-factor product, or for whom use of a non-factor product is a possibility, OR\n2. Females of any age, with confirmed congenital hemophilia A or B carrier status with genetic mutational analysis and any factor level.\n\nExclusion Criteria\n\n1. Presence of any known bleeding disorder other than congenital hemophilia A or B\n2. Presence of concurrent hemophilia and a second hemostatic defect (low von Willebrand Factor (vWF) without vWD diagnosis is not excluded)\n3. Unable or unwilling to comply with the study arm protocol.\n\nNonacog beta pegol (Rebinyn®) Module\n\nInclusion Criteria:\n\n1. Has provided signed written consent for the nonacog beta pegol (Rebinyn®)Module before any study-related activities.\n2. Male participants, at any age with hemophilia B, naïve or minimally exposed (up to 3 EDs) to nonacog beta pegol treatment at time of study enrollment. Additional doses may be allowable per ATHN Transcends PI approval.\n3. Decision to initiate continuous prophylaxis treatment with commercially available nonacog beta pegol has been made by the participant(s)\u002FLegally Authorized Representative(s) (LAR(s)) and the treating physician before and independently from the decision to include the participant in this study.\n\nExclusion Criteria:\n\n1. Previous participation in this study. Participation is defined as having given informed consent in this study.\n2. Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation, including a diagnosis or suspicion of attention deficit hyperactivity disorder (ADHD) or autism spectrum disorder (ASD) per the discretion of the Principal Investigator.\n3. Known or suspected hypersensitivity to nonacog beta pegol or related products.\n4. Clinical suspicion or presence of FIX inhibitor at time of inclusion.\n5. Inability or unwillingness to undergo neurological assessment\u002Fstructured developmental history.\n\nEmicizumab (Hemlibra®) Module\n\nInclusion Criteria:\n\n1. Participant currently treated with emicizumab (Hemlibra®)\n2. Currently enrolled in the Hemophilia Natural History Arm of ATHN Transcends\n\nExclusion Criteria:\n\n1\\. Unable or unwilling to comply with the protocol\n\nDistress Module\n\nInclusion Criteria:\n\n1. Congenital hemophilia A or B of any severity with or without inhibitors receiving a current therapy, a non-factor product, or for whom use of a non-factor product is a possibility\n2. Age 18 years of age or older\n3. English speaking\n\nExclusion Criteria:\n\n1. Presence of any known bleeding disorder other than congenital hemophilia A or B;\n2. Presence of concurrent hemophilia and a second hemostatic defect (low von Willebrand Factor (vWF) without vWD diagnosis is not excluded); and\n3. Unable or unwilling to comply with the study arm protocol\n\nHemophilia Gene Therapy Outcomes Arm\n\nInclusion Criteria\n\n1. Hemophilia A or B of any severity with or without inhibitors having received or will receive a hemophilia gene transfer product in the next 6 months.\n2. Age 18 years and older.\n3. Able to give informed consent.\n\nExclusion Criteria None\n\nEtranacogene dezaparvovec (HEMGENIX®) Module\n\nInclusion Criteria:\n\nEtranacogene dezaparvovec (HEMGENIX®) Cohort\n\n1. Age 18 years of age or older\n2. Treatment with commercial etranacogene dezaparvovec (HEMGENIX®)\n3. Have provided signed written informed consent within 3 months before or within 6 months after etranacogene dezaparvovec (HEMGENIX®) treatment, or within 6 months of when the study is initiated at the treating site.\n\nFIX Prophylaxis Cohort\n\n1. Age 18 years of age or older\n2. Treatment with FIX prophylaxis therapy\n3. Has provided signed written consent at any time for ATHN Transcends Study\n\nExclusion Criteria, both cohorts:\n\n1\\. Have been treated with etranacogene dezaparvovec in a clinical trial prior to commercial availability. These patients are still eligible for enrollment in the Gene Therapy Outcomes Arm, and their data may be collected for separate analysis.\n\nCongenital Platelet Disorders Arm\n\nInclusion Criteria\n\n1. Platelet adhesion defect\n\n   1. Bernard Soulier syndrome (Defective GPIb-IX-V receptor, impaired adhesion to vWF)\n   2. Velocardio-facial syndrome\u002FDiGeorge syndrome (Defective GPIb-IX-V receptor)\n   3. Platelet type vWD (Defective GPIb-IX-V, gain of function interaction between vWF-GP1bα)\n2. Platelet aggregation defect\n\n   1. Glanzmann thrombasthenia (Defective integrin αIIbβ3 (GPIIb\u002FIIIa)\n   2. Platelet aggregation defect, NOS\n3. Agonist receptor defects\n\n   1. Epinephrine\n   2. ADP\n   3. Collagen\n   4. Thromboxane A2\n4. Platelet signaling defects\n\n   1. Cyclooxygenase deficiency (PTGS1 mutation)\n   2. Phospholipase A2 deficiency\n   3. Thromboxane synthase deficiency (TBXAS1 mutation)\n   4. G protein activation defect (GNAS mutation)\n   5. Scott syndrome (defect in phosphatidyl serine translocation)\n5. Platelet Granule disorders\n\n   1. Dense granule storage pool disorder\n\n      * Hermansky Pudlak syndrome\n      * Chediak Higashi syndrome\n      * Griscelli syndrome\n   2. Alpha granule storage pool disorder\n\n      * Grey platelet syndrome\n      * Arthrogryposis-Renal Dysfunction-Cholestasis (ARC) syndrome\n      * Quebec platelet disorder\n      * Paris-Trousseau syndrome\n   3. Combined alpha delta granule deficiency\n6. Platelet cytoskeletal structure defects\n\n   1. Wiskott Aldrich syndrome\n   2. MYH9 associated disorders (myosin heavy chain)\n\n      * May Hegglin syndrome\n      * Fechtner syndrome\n      * Sebastian syndrome\n      * Epstein syndrome\n   3. Other mutations\n\n      * FLNA mutations (Filamin)\n      * DIAPH1 (Actin and microtubules)\n      * ACTN1 (alpha actinin)\n      * TPM4 (tropomyosin)\n      * TUBB1 (beta tubulin)\n7. Other Congenital thrombocytopenias\n\n   1. Familial platelet disorders and predisposition to AML (RUNX1)\n   2. X linked thrombocytopenia with dyserythropoiesis (GATA1)\n   3. Congenital amegakaryocytic thrombocytopenia (MPL)\n\nExclusion Criteria\n\n1. Diagnosis of von Willebrand Disease (Meeting the definition of vWD or low vWF per most recent international guidelines)\n2. Diagnosis of Hemophilia A or Hemophilia B (Factor VIII or IX ≤ 40%)\n\nGlanzmann Thrombasthenia (GT) Module\n\nInclusion Criteria\n\n1. Participant has signed the informed consent\u002Fassent form\n2. Participant has flow cytometry or aggregometry or genetics confirmed GT\n3. Participant is willing to perform study procedures, including daily bleed tracking for 3 months and further if requested\n4. Participants are 2 years or older at time of consent\n\nExclusion Criteria None","ALL",{"count":18,"type":19},3000,"ESTIMATED","OBSERVATIONAL","In parallel with the growth of ATHN's clinical studies, the number of new therapies for all blood disorders is increasing significantly. Some of the recently FDA-approved therapies for congenital and acquired hematologic conditions have not yet demonstrated long-term safety and effectiveness beyond the pivotal trials that led to their approval. In addition, results from well controlled, pivotal studies often cannot be replicated once a therapy has been approved for general use.2,3,4,5\n\nIn 2019 alone, the FDA has issued approvals for 24 new therapies for congenital and acquired hematologic conditions.6 In addition, almost 10,000 new studies for hematologic diseases are currently registered on www.clinicaltrials.gov.7\n\nWith this increase in potential new therapies possible, it is imperative that clinicians and clinical researchers in the field of non-neoplastic hematology have a uniform, secure, unbiased, and enduring method to collect long-term safety and efficacy data. As emphasized in a recently published review, accurate, uniform and quality national data collection is critical in clinical research, particularly for longitudinal cohort studies covering a lifetime of biologic risk.8",[23,24,25,26,27,28,29,30,31,32,33,34,35],"Hematologic Disorder","Bleeding Disorder","Connective Tissue Disorder","Hemophilia","Thrombosis","Von Willebrand Diseases","Thrombophilia","Rare Bleeding Disorder","Platelet Disorder","Factor IX Deficiency","Factor VIII Deficiency","Thalassemia","Sickle Cell Disease","RECRUITING","2026-07-10",{"date":39,"type":40},"2026-07-13","ACTUAL",{"date":42,"type":40},"2020-09-30",{"date":44,"type":19},"2035-12",{"name":46,"class":47},"American Thrombosis and Hemostasis Network","NETWORK",71,{"id":50,"slug":51,"hasResults":11,"nctId":52,"briefTitle":53,"officialTitle":54,"acronym":55,"eligibilityCriteria":56,"healthyVolunteers":11,"sex":57,"minAge":4,"maxAge":58,"enrollmentInfo":59,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":61,"conditions":62,"keywords":64,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":70,"lastUpdatePostDateStruct":71,"startDateStruct":73,"completionDateStruct":75,"leadSponsor":77,"locationsCount":80},"100573027","hormonal-management-of-heavy-menstrual-bleeding-in-adolescents-100573027","NCT06740929","Hormonal Management of Heavy Menstrual Bleeding in Adolescents","Hormonal Management of Heavy Menstrual Bleeding in Adolescents and Young Adults With and Without a Bleeding Disorder: a Prospective Observational Cohort Study","JAdoRE","Inclusion Criteria:\n\n* Adolescents and young adults aged \\\u003C 26yo\n* Presenting to the gynecology consultation at HUG\n* Diagnosed with heavy menstrual bleeding (PBAC \\> 100)\n* Willing to initiate a hormonal medication to manage heavy menstrual bleeding\n\nExclusion Criteria:\n\n* Pregnancy or planning to become pregnant during the study period\n* Already using hormonal medication for menstrual management and\u002For contraception\n* Known anatomical uterine pathology\n* Using anticoagulant or antiaggregant agents","FEMALE","25 Years",{"count":60,"type":19},163,"The goal of this study is to evaluate whether hormonal medications (such as contraceptive pill, patch, injectable progestins and hormonal intra-uterine device) have similar efficacy at managing heavy menstrual bleeding in adolescents with a bleeding disorder compared to those without. We also aim to assess the quality of life and hemoglobin and iron levels in this population.",[63,24],"Heavy Menstrual Bleeding",[65,66,67,68,69],"heavy menstrual bleeding","bleeding disorder","adolescent","contraception","menstrual management","2026-05-11",{"date":72,"type":40},"2026-05-12",{"date":74,"type":40},"2024-12-17",{"date":76,"type":19},"2027-12",{"name":78,"class":79},"University Hospital, Geneva","OTHER",1,{"id":82,"slug":83,"hasResults":11,"nctId":84,"briefTitle":85,"officialTitle":86,"acronym":87,"eligibilityCriteria":88,"healthyVolunteers":89,"sex":16,"minAge":90,"maxAge":91,"enrollmentInfo":92,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":94,"conditions":95,"keywords":96,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":102,"lastUpdatePostDateStruct":103,"startDateStruct":105,"completionDateStruct":107,"leadSponsor":109,"locationsCount":110},"100494096","bleeding-of-unknown-cause-a-swiss-case-control-study-100494096","NCT05713734","Bleeding of Unknown Cause: a Swiss Case-control Study","Bleeding of Unknown Cause, Insight Into a Multifactorial Bleeding Disorder: a Swiss Case-control Study","SWISS-BUC","Inclusion Criteria:\n\n* Man with ISTH BAT\\>3. If the calculated score includes a surgical bleeding, then two other items of the score should be \\>0\n* Woman with ISTH BAT \\>5 points. If the calculated score includes a surgical or a postpartum bleeding, then two other items of the score should be \\>0\n\nExclusion Criteria:\n\n* Ongoing pregnancy\n* Intake of antithrombotic treatment or non-steroidal anti-inflammatory drugs for least 10 days at time of blood collection\n* Intake of antifibrinolytic or blood product administration (factor concentrate, frozen fresh plasma, prothrombin complex concentrate) for least 14 days before blood collection\n* Active cancer (defined as cancer diagnosis within the last five years or treatment within the two last years before study inclusion)\n* Active autoimmune disease\n* Active chronic inflammatory disease\n* Severe liver disease (cirrhosis \\> Child A)\n* Renal insufficiency stage 3\n* Active or recent infection (within the last 30 days)\n* Recent hospitalization (\\\u003C3 months)\n* Recent surgery (\\\u003C3 months)\n* Recent trauma requiring medical intervention (\\\u003C3 months)",true,"16 Years","65 Years",{"count":93,"type":19},400,"The goal of this prospective cohort is to identified specific biological patterns in patients with a bleeding of unknown cause and to study the specific mechanisms of the bleeding disorder for each subset of patients.",[24],[97,98,99,100,101],"Bleeding","Hemostatics","Fibrin","Fibrinolysis","Thrombin","2026-04-28",{"date":104,"type":40},"2026-04-29",{"date":106,"type":40},"2023-04-01",{"date":108,"type":19},"2027-04-01",{"name":78,"class":79},7,{"id":112,"slug":113,"hasResults":11,"nctId":114,"briefTitle":115,"officialTitle":116,"acronym":4,"eligibilityCriteria":117,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":118,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":120,"conditions":121,"keywords":126,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":132,"lastUpdatePostDateStruct":133,"startDateStruct":135,"completionDateStruct":137,"leadSponsor":139,"locationsCount":80},"100579147","athndataset-registry-100579147","NCT06820515","ATHNdataset Registry","American Thrombosis and Hemostasis Network ATHNdataset Registry","Inclusion Criteria:\n\n* Any participant evaluated for or the potential to have a blood disorder who has an encounter with an ATHN Affiliate.\n* Participants of any age.\n* Participant is able to provide consent or assent; a Legally Authorized Representative (LAR) may provide consent on a participant's behalf if a participant is unable to provide self-consent\n\nExclusion Criteria:\n\n* Any participant unable to provide consent or assent to participate in the ATHNdataset",{"count":119,"type":19},200000,"The Hemophilia Treatment Center (HTC) where you receive care is working with The American Thrombosis and Hemostasis Network (ATHN) to look at the quality of life of people with blood disorders and problems.\n\nDoctors, scientists, policymakers, and other health care providers need a large amount of information from a lot of people to answer scientific, public health, and policy questions about better ways to treat blood disorders. They will use the information from the ATHNdataset to answer these questions.",[26,27,122,123,35,124,24,125,28],"Hemophilia A","Hemophilia B","Glanzmann Thrombasthenia","Blood Disorder",[127,128,129,130,66,131],"bleed event","bleed treatments","adverse events","joint bleed","bleeding symptoms","2026-04-16",{"date":134,"type":40},"2026-04-21",{"date":136,"type":40},"2024-10-25",{"date":138,"type":19},"2055-10-31",{"name":46,"class":47},{"id":141,"slug":142,"hasResults":11,"nctId":143,"briefTitle":144,"officialTitle":145,"acronym":146,"eligibilityCriteria":147,"healthyVolunteers":89,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":148,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":150,"conditions":151,"keywords":163,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":170,"lastUpdatePostDateStruct":171,"startDateStruct":173,"completionDateStruct":175,"leadSponsor":177,"locationsCount":80},"100624813","hemophilia-a-research-program-100624813","NCT07414511","Hemophilia A Research Program","Hemophilia A Research Program (HARP): An Observational Intergenerational Cohort Study of Hemophilia A and Factor VIII Immunogenicity","HARP","Maternal Inclusion Criteria:\n\nPregnant individuals who meet the following criteria are eligible for enrollment as study participants:\n\n* Currently pregnant and prior to 37 weeks gestation\n* Known to have or at-risk of having a severe hemophilia A genotype\n* Pregnant with at least one fetus at-risk of inheriting severe hemophilia A\n* Ability to understand and willingness to provide informed consent\n* 18 years of age or older\n\nBefore the 38th week of pregnancy, enrolled participants must meet all the following criteria to continue to remain in the study:\n\n* The pregnant mother has a severe hemophilia A genotype.\n* A fetus is determined to have a \\>\u002F= 25% risk of inheriting severe hemophilia A, or prenatal testing indicates a fetus is affected by severe hemophilia A.\n* No other discontinuation criteria have been identified.\n\nPediatric Continuation \u002F Inclusion Criteria:\n\nEligibility of the child to continue is assessed by age 8 weeks. Mother-child pairs in which a child meets the following criteria will remain in the study:\n\n* Severe hemophilia A defined by a baseline FVIII:C \\\u003C 0.01 IU\u002FmL (or FVIII:C \\\u003C 1%) or a genotype predicted to cause severe hemophilia A\n* Born to a mother participating in the study\n\nThereafter, mothers and their children will continue in the study as long as no new discontinuation criteria occur.\n\nInclusion Criteria for Blood Relatives:\n\nBlood relatives of the child may be offered participation if one of the following criteria are met:\n\n* First-degree blood relatives (e.g., father, sibling) of the child\n* Second-degree blood relatives (e.g., aunt, uncle, grandparent, half-sibling) of the child\n* Any more distant male or female blood relative whose data or samples may be informative for the planned genetic studies of hemophilia and inhibitors\n\nExclusion\u002FDiscontinuation Criteria:\n\nMaternal: For the pregnant person, exclusion or discontinuation criteria are as follows:\n\n* Genetic testing is negative for a severe hemophilia A genotype\n* Prenatal clinical diagnostic testing that indicates there is no fetus affected with severe hemophilia A\n* Presence of another clinically significant bleeding disorder\n* Participation in another study for which any blood collection total would exceed safety limits defined in this study\n* Will deliver outside the United States or plans for regular pediatric care for the child to be delivered outside the United States\n* Is a prisoner\n* Any other reason that, in the opinion of the investigator, would render the individual unsuitable for participation in the study\n* Inability for study team to obtain translated study documents in time for participation if participant is not fluent in English\n\nPediatric: For the child, discontinuation criteria are as follows:\n\n* Infant does not have severe hemophilia A defined by a baseline FVIII:C \\\u003C 0.01 IU\u002FmL (or FVIII:C \\\u003C 1%) or does not have a genotype predicted to cause severe hemophilia A\n* Mother or child did not have minimal required study samples or data collected before birth, around the time of delivery, or in the neonatal period\n* Child has another clinically significant bleeding disorder\n* Child has a clinically severe immune disorder\n* Participation in another study for which any blood collection total would exceed safety limits defined in this study\n* Any other reason that, in the opinion of the investigator, would render the individual unsuitable for participation in the study",{"count":149,"type":19},500,"This study longitudinally observes the intergenerational (mother-child) continuum in hemophilia A from pregnancy through early childhood. Because the study follows mother-child pairs, the study includes both a maternal cohort and a pediatric cohort. Each cohort has a primary goal: for the mother with a severe hemophilia genotype, the overarching primary goal is to understand the risks for pregnancy-associated bleeding and postpartum hemorrhage (PPH); for the child, the overarching primary goal is to understand the risks, timing, and circumstances of development of anti-FVIII antibodies. From a longitudinal perspective, risks for both bleeding in the mother and anti-FVIII antibody development in the child are expected to be influenced over time by genetic and environmental factors that begin early in (or before) pregnancy. Enrollment of blood relatives is offered to improve power to better understand inherited contributions to bleeding and inhibitor development in the mother-baby pairs.",[122,152,153,154,155,156,157,158,159,24,160,161,162],"Hemophilia A, Severe","Factor VIII (FVIII)","FVIII Deficiency","Carrier of Hemophilia A","Inhibitors","Pregnancy","Maternal Blood Loss","Pregnancy Complications","Hemorrhage, Postpartum","Alloimmunization","X-Linked",[157,164,97,24,165,26,122,166,167,168,169],"Inhibitor","Maternal Child Health","Hemophilia A Carrier","Hemophilia A Symptomatic Carrier","Coagulation","Factor VIII","2026-02-09",{"date":172,"type":40},"2026-02-17",{"date":174,"type":40},"2024-07-31",{"date":176,"type":19},"2029-08",{"name":178,"class":79},"University of Washington",{"id":180,"slug":181,"hasResults":11,"nctId":182,"briefTitle":183,"officialTitle":184,"acronym":185,"eligibilityCriteria":186,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":187,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":189,"conditions":190,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":170,"lastUpdatePostDateStruct":204,"startDateStruct":206,"completionDateStruct":208,"leadSponsor":210,"locationsCount":80},"100412026","national-longitudinal-cohort-of-hematological-diseases-100412026","NCT04645199","National Longitudinal Cohort of Hematological Diseases","National Longitudinal Cohort of Hematological Diseases (NICHE)","NICHE","Inclusion Criteria:\n\n* Patients who were diagnosed with acute myeloid leukemia, multiple myeloma, hemophilia, aplastic anemia, leukemia, myelodysplastic syndrome, lymphoma, bleeding disorders or received bone marrow transplantation in the investigating hospitals from January 1, 2020.\n\nExclusion Criteria:\n\n* Long-term follow-up information for patients is not available for any reason, such as not being available or having a serious concomitant disease.\n* Alcohol and drug addictions affect their ability to comply with study requirements.\n* According to the investigator, there are conditions that may endanger the patient's safety or affect his\u002Fher compliance.",{"count":188,"type":19},2300,"Background Hematological diseases are disorders of the blood and hematopoietic organs. The current hematological cohorts are mostly based on single-center or multi-center cases, or cohorts with limited sample size in China. There is a lack of comprehensive and large-scale prospective cohort studies in hematology. The purpose of this study is to analyze the incidence and risk factors of major blood diseases, the treatment methods, prognosis and medical expenses of these patients in China.\n\nMethod The study will include patients diagnosed with acute myeloid leukemia, multiple myeloma, hemophilia, aplastic anemia, leukemia, myelodysplastic syndrome, lymphoma, bleeding disorders, autoimmune hemolytic anemia, large granular lymphocyte leukemia, essential thrombocythemia, blood infection or received bone marrow transplantation in the investigating hospitals from January 1, 2020, and collect basic information, diagnostic and treatment information, prognosis information, as well as medical expense information from medical records. In its current form, the NICHE registry incorporates historical data (collected from 2000) and is systematically collecting prospective data in two phases with broadening reach, and prospectively follow-up to collect the prognosis information.",[191,192,26,122,123,193,194,195,196,197,24,198,199,200,201,202,203],"Multiple Myeloma","Acute Myeloid Leukemia","Myelodysplastic Syndrome","MDS","Lymphoma","Leukemia","Aplastic Anemia","Bone Marrow Transplantation","Blood Disease Infection","Autoimmune Hemolytic Anemia, AIHA","Essential Thrombocythemia, ET","Large Granular Lymphocyte Leukemia, LGLL","Paroxysmal Nocturnal Hemoglobinuria, PNH",{"date":205,"type":40},"2026-02-12",{"date":207,"type":40},"2020-12-01",{"date":209,"type":19},"2030-12-01",{"name":211,"class":79},"Institute of Hematology & Blood Diseases Hospital, China",{"id":213,"slug":214,"hasResults":11,"nctId":215,"briefTitle":216,"officialTitle":217,"acronym":4,"eligibilityCriteria":218,"healthyVolunteers":11,"sex":16,"minAge":219,"maxAge":4,"enrollmentInfo":220,"targetDuration":4,"studyType":222,"phases":223,"briefSummary":225,"conditions":226,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":227,"lastUpdatePostDateStruct":228,"startDateStruct":230,"completionDateStruct":232,"leadSponsor":234,"locationsCount":80},"100525179","phase-1-recombinant-vwf-concentrate-and-ecmo-100525179","NCT06118372","Recombinant vWF Concentrate and ECMO","Safety and Tolerability of Recombinant Von Willebrand Factor Concentrate in Adult ECMO Patients With Major Bleeding: A Phase I Study","Inclusion Criteria:\n\n1. Adult patients (18 years or greater)\n2. On extracorporeal membrane oxygenation\n3. Major bleeding defined by CTCAE class 3 or greater\n4. Off systemic anticoagulation for at least 4 hours\n\nExclusion Criteria:\n\n1. Platelet count less than 40 x 109\u002FL\n2. International normalized ratio\\> 2.0\n3. Fibrinogen less than 150 mg\u002FdL\n4. Current participation in another clinical trial (interventional)\n5. Heparin induced thrombocytopenia (active)\n6. Acute liver failure, as indicated by bilirubin \\>20 mg\u002FdL or new onset hepatic encephalopathy\n7. Patient or legally authorized representative unable to give informed consent\n8. Allergy to recombinant von Willebrand Factor or any component of the product based on prior exposure\n9. Of childbearing age and positive pregnancy test during the same hospital admission, a pregnancy test will be mandatory for all women of child-bearing age\n10. Known congenital or acquired thrombophilia\n11. History of deep venous thrombosis, pulmonary embolism, circuit thrombosis, disseminated intravascular coagulation (DIC), ischemic stroke, ST elevation myocardial infarction (STEMI), or arterial thrombosis in the last 3 months.\n12. History of hypersensitivity to vWF concentrate\n13. Known history of vWF antibodies","18 Years",{"count":221,"type":19},12,"INTERVENTIONAL",[224],"PHASE1","Adult patients on extracoporeal membrane oxygenation (ECMO) frequently experience bleeding, which is in part caused by acquired von Willebrand syndrome (vWS). Prior in vitro studies have shown that the addition of recombinant von Willebrand Factor (vWF) to ECMO patient blood samples, normalizes platelet adhesion and thrombus formation. This study is a phase I study, where adult ECMO patients with refractory bleeding will be treated with recombinant vWF a single time. The primary objectives are to evaluate the safety, tolerability, and pharmacokinetics of recombinant vWF in adult ECMO patients.",[24],"2026-02-02",{"date":229,"type":40},"2026-02-05",{"date":231,"type":40},"2024-10-03",{"date":233,"type":19},"2027-02-28",{"name":235,"class":79},"University of Virginia",{"id":237,"slug":4,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":238,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":21,"conditions":239,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":240,"lastUpdatePostDateStruct":241,"startDateStruct":243,"completionDateStruct":244,"leadSponsor":245,"locationsCount":48},"100393100",{"count":18,"type":19},[23,24,25,26,27,28,29,30,31,32,33,34,35],"2026-01-09",{"date":242,"type":40},"2026-01-12",{"date":42,"type":40},{"date":44,"type":19},{"name":46,"class":47},{"id":247,"slug":248,"hasResults":11,"nctId":249,"briefTitle":250,"officialTitle":250,"acronym":251,"eligibilityCriteria":252,"healthyVolunteers":11,"sex":57,"minAge":253,"maxAge":254,"enrollmentInfo":255,"targetDuration":257,"studyType":20,"phases":4,"briefSummary":258,"conditions":259,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":260,"lastUpdatePostDateStruct":261,"startDateStruct":263,"completionDateStruct":265,"leadSponsor":267,"locationsCount":269},"100509672","heavy-menstrual-bleeding-progestin-treatment-in-bleeding-disorders-study-100509672","NCT05916469","Heavy Menstrual Bleeding Progestin Treatment in Bleeding Disorders Study","HMB-BD","Inclusion Criteria:\n\n* Post-menarcheal\n* Ages 10-24\n* Decision to initiate a trial of progestin therapy with either LNG-IUD or NETA\n* Able to provide assent and written informed consent by one parent (ages \\\u003C18) or written informed consent (age 18+)\n* Must meet trial criteria for heavy menstrual bleeding\n* Must meet trial criteria for a bleeding disorder or have minimum workup to rule out bleeding disorder\n\nExclusion Criteria:\n\n* Pregnant or seeking pregnancy","10 Years","24 Years",{"count":256,"type":19},300,"5 Years","The goal of this multicenter prospective observational study and registry of U.S. adolescents and young adults with heritable bleeding disorders is to determine the bleeding outcomes, satisfaction, hemostatic parameter changes, and patient reported quality of life after 6 months of use of either of two commonly used hormonal treatments for menstrual suppression - levonorgestrel intrauterine device (LNG-IUD) and norethindrone acetate (NETA). Under this application the study will compare the two treatments and compare outcomes after LNG-IUD treatment results to a control group without a bleeding disorder, with the goal of determining the benefits and expected outcomes of these treatment options for this population.",[63,24,28],"2025-12-09",{"date":262,"type":40},"2025-12-17",{"date":264,"type":40},"2025-09-24",{"date":266,"type":19},"2029-06",{"name":268,"class":79},"Oregon Health and Science University",9,{"id":271,"slug":272,"hasResults":11,"nctId":273,"briefTitle":274,"officialTitle":275,"acronym":276,"eligibilityCriteria":277,"healthyVolunteers":11,"sex":16,"minAge":278,"maxAge":4,"enrollmentInfo":279,"targetDuration":4,"studyType":222,"phases":281,"briefSummary":283,"conditions":284,"keywords":285,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":287,"lastUpdatePostDateStruct":288,"startDateStruct":290,"completionDateStruct":292,"leadSponsor":294,"locationsCount":296},"100572660","early-phase-1-early-genomic-testing-for-inherited-bleeding-disorders-100572660","NCT06736158","Early Genomic Testing for Inherited Bleeding Disorders","Early Genomic Testing for Inherited Bleeding Disorders in Patients Without a Diagnosis After First Line Testing: a Randomized Controlled Trial","GT4BD","Inclusion Criteria:\n\n* New patient referred for abnormal bleeding.\n* Hemostasis expert clinician determined abnormal bleeding history AND family history of bleeding\n* OR no family history of bleeding but hemostasis expert clinician determined severe bleeding history.\n\nExclusion Criteria:\n\n* Prior diagnosis of an inherited bleeding disorder.\n* Acquired cause of bleeding (i.e., medication known to cause bleeding, significant renal or hepatic disease)","12 Years",{"count":280,"type":19},212,[282],"EARLY_PHASE1","The investigators aim to test the introduction of genomic testing early in the diagnostic pathway for inherited bleeding disorders in patients who have not received a diagnosis after first-line testing.\n\nThe goal of this clinical trial is to test the introduction of genomic testing early in the diagnostic pathway for patients referred to Hematology for a suspected inherited bleeding disorder. The main questions it aims to answer are:\n\n1. Does adding early genomic testing increase the number of patients who are diagnosed?\n2. Does adding early genomic testing decrease the overall time to diagnosis?\n3. Is it cost-effective to include early genomic testing in the diagnostic pathway?\n\nThe investigators will compare with a control group of participants who are receiving standard care (no early genomic testing).\n\nParticipants will randomized to a standardized diagnostic testing plus early genomic testing group or to the standardized diagnostic testing group only (with the possibility of being offered genomic testing after 1 year in the study).",[24],[286,66],"blood coagulation disorders","2025-07-28",{"date":289,"type":40},"2025-07-31",{"date":291,"type":40},"2025-05-31",{"date":293,"type":19},"2027-04-30",{"name":295,"class":79},"Queen's University",3,{"id":298,"slug":299,"hasResults":11,"nctId":300,"briefTitle":301,"officialTitle":302,"acronym":303,"eligibilityCriteria":304,"healthyVolunteers":11,"sex":16,"minAge":219,"maxAge":4,"enrollmentInfo":305,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":307,"conditions":308,"keywords":313,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":316,"lastUpdatePostDateStruct":317,"startDateStruct":319,"completionDateStruct":321,"leadSponsor":323,"locationsCount":80},"100564929","personalized-viscoelastic-testing-guided-bleeding-management-in-liver-surgery-neurosurgery-and-obstetrics-100564929","NCT06635564","Personalized ViscoElastic Testing-guided Bleeding Management In Liver Surgery, Neurosurgery and Obstetrics","Personalized ViscoElastic Testing-guided Bleeding Management In Liver Surgery, Neurosurgery and Obstetrics - a Prospective Comparison of the Novel ClotPro With ROTEM and TEG","VETILNO","Inclusion Criteria:\n\n1. Vulnerable patient cohorts\n\n   * Patients undergoing elective liver surgery defined as one of the following invasive procedures:\n\n     * Liver resection (anatomic or non-anatomic segmental resection, right or left hepatectomy, right or left extended hepatectomy),\n     * Orthotopic liver transplantation,\n   * Pregnant women undergoing an elective caesarean section, and\n   * Patients undergoing an elective intracranial neurosurgery.\n2. Written informed consent\n\nExclusion Criteria:\n\nnone",{"count":306,"type":19},240,"The ClotPro analyzer is a new generation viscoelastic analyzer for the in vitro assessment of blood coagulation. This study aims to assess the agreement of ClotPro 6.0, ROTEM delta, and TEG 6s in three distinct cohorts: i) patients with liver disease undergoing liver surgery, ii) pregnant women undergoing elective cesarean section, and iii) patients undergoing elective intracranial neurosurgery. Further coagulation tests will be performed (standard laboratory coagulation tests, thrombin and plasmin generation tests) in an exploratory fashion to compare them with viscoelastic test results. The obtained test results will not result in any diagnostic or therapeutic consequences for patients included in this study.",[309,310,311,24,312],"Thrombelastography","Liver Transplant","Postpartum Hemorrhage","Intracranial Hemorrhages",[314,315],"Viscoelastic testing","personalized bleeding management","2025-05-20",{"date":318,"type":40},"2025-05-23",{"date":320,"type":40},"2024-07-01",{"date":322,"type":19},"2026-07-31",{"name":324,"class":79},"Medical University of Vienna",{"id":326,"slug":327,"hasResults":11,"nctId":328,"briefTitle":329,"officialTitle":329,"acronym":4,"eligibilityCriteria":330,"healthyVolunteers":89,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":331,"targetDuration":257,"studyType":20,"phases":4,"briefSummary":332,"conditions":333,"keywords":345,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":347,"lastUpdatePostDateStruct":348,"startDateStruct":350,"completionDateStruct":352,"leadSponsor":354,"locationsCount":356},"100572008","longitudinal-cohort-of-thrombosis-and-hemostasis-diseases-100572008","NCT06727669","Longitudinal Cohort of Thrombosis and Hemostasis Diseases","Inclusion Criteria:\n\n* Patients who were diagnosed as thrombosis and hemostasis diseases.\n\nExclusion Criteria:\n\n* Long-term follow-up information for patients is not available for any reason, such as not being available or having a serious concomitant disease.\n* Patients with alcohol and drug addictions or mental illness affect their ability to comply with study requirements.\n* According to the investigator, there are conditions that may endanger the patient's safety or affect his\u002Fher compliance.",{"count":18,"type":19},"This is a multicenter, prospective, longitudinal, observational cohort study to investigate thrombosis and hemostasis diseases in Chinese patients. This study will collect basic information, diagnostic and treatment information, as well as medical expense information of patients from medical records.The incidence and risk factors of thrombosis and hemostasis diseases, the treatment methods, prognosis and medical expenses of these patients in China will be analyzed. The study will use questionnaire to measure the exposure of patients, and prospectively follow-up to collect the prognosis information.",[334,335,336,337,29,338,339,340,341,122,123,342,343,344,24,27],"Immune Thrombocytopenia","Thrombotic Thrombocytopenic Purpura","Hemophilia A, Acquired","Disseminated Intravascular Coagulation","Deep Vein Thrombosis","Pulmonary Embolism","Thrombotic Microangiopathies","Coagulation Factor Deficiency","Hemophilia B, Acquired","Platelet Dysfunction","Arterial Thromboembolism",[27,346],"Hemostasis","2024-12-05",{"date":349,"type":40},"2024-12-11",{"date":351,"type":40},"2024-11-01",{"date":353,"type":19},"2030-12-31",{"name":355,"class":79},"Peking University People's Hospital",5,{"id":358,"slug":359,"hasResults":11,"nctId":360,"briefTitle":361,"officialTitle":361,"acronym":362,"eligibilityCriteria":363,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":364,"enrollmentInfo":365,"targetDuration":367,"studyType":20,"phases":4,"briefSummary":368,"conditions":369,"keywords":379,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":386,"lastUpdatePostDateStruct":387,"startDateStruct":389,"completionDateStruct":391,"leadSponsor":393,"locationsCount":80},"100535343","european-rare-blood-disorders-platform-enrol-100535343","NCT06250595","European Rare Blood Disorders Platform (ENROL)","ENROL","Inclusion Criteria:\n\n* Patients must meet all of the following criteria to be included in the ENROL Registry\n* Age from 0-100, both female and male\n* Diagnosed as RHDs according to ORPHANET classification\n* Able and willing to provide written informed consent (patient or legal representative for minors) if needed according to national legislation.\n\nExclusion Criteria:\n\n* Patients diagnosed as traits or trait conditions for other recessive RHDs","100 Years",{"count":366,"type":19},37090,"15 Years","ENROL, the European Rare Blood Disorders Platform has been conceived in the core of ERN-EuroBloodNet as an umbrella for both new and already existing registries on Rare Hematological Diseases (RHDs). ENROL aims at avoiding fragmentation of data by promoting the standards for patient registries' interoperability released by the EU RD platform.\n\nENROL's principle is to maximize public benefit from data on RHDs opened up through the platform with the only restriction needed to guarantee patient rights and confidentiality, in agreement with EU regulations for cross-border sharing of personal data.\n\nAccordingly, ENROL will map the EU-level demographics, survival rates, diagnosis methods, genetic information, main clinical manifestations, and treatments in order to obtain epidemiological figures and identify trial cohorts for basic and clinical research. To this aim, ENROL will connect and facilitate the upgrading of existing RHD registries, while promoting the building of new ones when \u002F where lacking. Target-driven actions will be carried out in collaboration with EURORDIS for educating patients and families about the benefits of enrolment in such registries, including different cultural and linguistic strategies.\n\nThe standardized collection and monitoring of disease-specific healthcare outcomes through the ENROL user-friendly platform will determine how specialized care is delivered, where are the gaps in diagnosis, care, or treatment and where best to allocate financial, technical, or human resources.\n\nMoreover, it will allow for promoting research, especially for those issues that remain unanswered or sub-optimally addressed by the scientific community; furthermore, it will allow promoting clinical trials for new drugs. ENROL will enable the generation of evidence for better healthcare for RHD patients in the EU as the ultimate goal.\n\nENROL officially started on 1st June 2020 with a duration of 36 months. ENROL is co-funded by the Health Programme of the European Union under the call for proposals HP-PJ-2019 on Rare disease registries for the European Reference Networks. GA number 947670",[370,371,24,372,373,374,375,196,376,34,377,378],"Anemia","Bone Marrow Failure","Iron Metabolism Disorders","Myeloma","Lymphoid Neoplasm","Myeloma, Malignant","Anemia, Sickle Cell","Blood Cancer","Red Cell Membrane and Enzyme Abnormalities",[370,371,380,381,382,383,384,196,385,34,35],"Bleeding disorder","Iron metabolism disorder","Myeloid","Lymphoid","Blood cancer","Red Cell membrane and Enzyme Abnormalities","2024-02-06",{"date":388,"type":40},"2024-02-09",{"date":390,"type":40},"2022-07-01",{"date":392,"type":19},"2037-07",{"name":394,"class":79},"Hospital Universitari Vall d'Hebron Research Institute"]