Consensus opinion on immune-mediated cytopenias after hematopoietic cell transplant for inherited metabolic disorders

Allogeneic hematopoietic stem cell transplantation for inherited metabolic disorders

“…In most cases with IMCs, the presence of antibodies against red blood cell lineage is easily documented, while it is less commonly detected in patients with thrombocytopenia or neutropenia. Gupta et al reported a proposal on the diagnosis and management of IMCs from a panel that included experts in HSCT for IMDs as well as experts in non-malignant disorders and transplant immunology [49]. In cytopenia involving the red blood cell lineage, namely, autoimmune hemolytic anemia (AIHA), the diagnostic criteria include (1) hemoglobin ≤ 7 g/dL or a drop in previously stable hemoglobin of more than 2 g/dL within 5 days without any other attributable cause and (2) the presence of red blood cell-directed antibody (DAT-positive).…”

Successful sequential therapy with rituximab and daratumumab for refractory post-transplant immune thrombocytopenic purpura

“…To the Editor, Immune-mediated cytopenias (IMCs), including immune thrombocytopenic purpura (ITP), autoimmune hemolytic anemia, and autoimmune neutropenia, have been newly highlighted as a significant complication after allogeneic hematopoietic cell transplantation (HCT), with a cumulative incidence of 2.1% to 52.6%. 1,2 Major risk factors for post-transplant IMC include younger recipient age at HCT, non-malignant disease, cord blood transplantation, unrelated donor, and a reduced-intensity conditioning (RIC) regimen. 1,2 Posttransplant IMCs are occasionally resistant to conventional first-line therapy, i.e., corticosteroid and/or intravenous immunoglobulin (IVIG) therapy in combination with supportive therapy, such as granulocyte colonystimulating factor, thrombopoietin receptor agonist (TPO-RA), and transfusion.…”

“…1,2 Major risk factors for post-transplant IMC include younger recipient age at HCT, non-malignant disease, cord blood transplantation, unrelated donor, and a reduced-intensity conditioning (RIC) regimen. 1,2 Posttransplant IMCs are occasionally resistant to conventional first-line therapy, i.e., corticosteroid and/or intravenous immunoglobulin (IVIG) therapy in combination with supportive therapy, such as granulocyte colonystimulating factor, thrombopoietin receptor agonist (TPO-RA), and transfusion. Rituximab, an anti-CD20 monoclonal antibody (mAb), mycophenolate mofetil, bortezomib, and daratumumab, an anti-CD38 mAb, have been used as second-line or subsequent therapies for refractory or recurrent cases.…”

“…Rituximab, an anti-CD20 monoclonal antibody (mAb), mycophenolate mofetil, bortezomib, and daratumumab, an anti-CD38 mAb, have been used as second-line or subsequent therapies for refractory or recurrent cases. [1][2][3][4][5] A 17-year-old boy with adrenoleukodystrophy underwent allogeneic bone marrow transplantation (BMT) from a human leukocyte antigen (HLA) 7/8 allele-matched unrelated donor using a RIC regimen consisting of fludarabine (125 mg/m 2 ), melphalan (140 mg/m 2 ), rabbit anti-thymocyte globulin (5 mg/kg), and total body irradiation of 4 Gy. Tacrolimus and short-term methotrexate were administered as prophylaxis for graft-versus-host disease (GVHD).…”

Successful sequential therapy with rituximab and daratumumab for refractory posttransplant immune thrombocytopenic purpura

“…reduced-intensity conditioning (RIC) regimen. 1,2 Posttransplant IMCs are occasionally resistant to conventional first-line therapy, that is, corticosteroid and/or intravenous immunoglobulin (IVIG) therapy in combination with supportive therapy, such as granulocyte colonystimulating factor, thrombopoietin receptor agonist (TPO-RA), and transfusion. Rituximab, an anti-CD20 monoclonal antibody (mAb), mycophenolate mofetil, bortezomib, and daratumumab, an anti-CD38 mAb, have been used as second-line or subsequent therapies for refractory or recurrent cases.…”