PTCy and and Ruxolitinib for GVHD Prophylaxis After HSCT With Thymoglobulin in Conditioning Regimen in Patients With Inborn Errors of Immunity

Hematopoietic stem cell transplantation (HSCT) is widely used in inborn errors of immunity (IEI), and risks of graft-versus-host disease (GVHD) remain high. Use of post-transplant cyclophosphamide (PTCY) for GVHD prophylaxis revolutionized the outcomes of HSCT from mismatched related donor (MMRD). Use of ruxolitinib for GVHD prophylaxis demonstrates promising results in adult patients. Another well-known option for GVHD prevention is antithymocyte globulin. To evaluate the efficacy of combination of thymoglobulin with PTCY and ruxolitinib for GVHD prophylaxis, conditioning regimen containing treosulfan 30-42 g/m2, fludarabine 150 mg/mg, and thiotepa 10 mg/kg or melphalan 140 mg/m2 and GVHD prophylaxis regimen containing cyclophosphamide 50 mg/kg for MMRD, 25 mg/kg for matched unrelated and related donors at days +3, 4 post-HSCT and ruxolitinib at dose 7 mg/m2 from day +5 after HSCT will be used in patients with IEI.

• Overall survival [Срок оценки: 1 year after HSCT]
• Cumulative incidence of acute graft versus host disease [Срок оценки: 1 year after HSCT]
• Cumulative incidence of chronic graft versus host disease [Срок оценки: 1 year after HSCT]
• Cumulative incidence of engraftment [Срок оценки: 100 days]
• Cumulative incidence of graft failure [Срок оценки: 1 year after HSCT]
• Incidence of early organ toxicity [Срок оценки: 100 days]
• Cumulative incidence of transplant related mortality [Срок оценки: 1 year after HSCT]
• Cumulative incidence of viral infections [Срок оценки: 1 year after HSCT]
• Investigation of the concentration of ruxolitinib in the blood To investigate the pharmacokinetics of ruxolitinib [Срок оценки: 1 month after HSCT]