Thrombocytopaenia

The goal of this clinical trial is to evaluate the efficacy and safety of sirolimus in patients with anti-phospholipid antibody-associated thrombocytopenia. In the 6-month randomized, double-blind, placebo-controlled phase, participants will receive either sirolimus 1 mg once daily or matching placebo. Participants will be followed at 2 weeks, 1 month, 3 months, and 6 months after enrollment. Participants who do not achieve the primary endpoint at 6 months may enter a 3-month open-label extension and receive sirolimus 1.5 mg once daily, with follow-up through month 9. The main question is whether sirolimus improves the overall response rate at 6 months compared with placebo. Primary outcome: \- Overall response rate at 6 months Secondary outcomes: * Overall response rate at 1 month and 3 months * Complete response rate at 6 months * Partial response rate at 6 months * Change in anti-phospholipid antibody titers at 6 months * Change in oral glucocorticoid dosage at 6 months Exploratory outcomes: * Proportion and reasons for early discontinuation during the double-blind phase * Among participants who do not achieve the primary endpoint at 6 months and enter the open-label extension, overall response rate, complete response rate, partial response rate, and safety outcomes after 3 months of open-label treatment

Romiplostim has demonstrated its efficacy and good tolerance in multiple indications. If the efficacy of Romiplostim is confirmed in this population, thrombocytopenic patients will be able to undergo cardiac surgery, which is generally life-saving, without prior thrombocytopenia. Patients included in the study will be hospitalized within 15 days of the first administration of the study treatment and will be closely monitored clinically and biologically for at least 10 days after surgery. The occurrence of an adverse event can therefore be quickly detected and managed. Collectively, "Patient Blood Management" strategies are mainly focused on the management of preoperative anemia. If positive, this study will enrich the therapeutic arsenal available for optimizing patients in preparation for major surgery. As no major collective complications are expected, the collective benefit/risk ratio also appears favorable.

The aim of this study is to analyze the effect of the storage time of platelet concentrates on the occurrence of bleeding events during prophylactic platelet transfusions in patients with hematological malignancies.