Dose-adapted post-transplant cyclophosphamide for HLA-haploidentical transplantation in Fanconi anemia
| Autor: | Lauri S Burroughs, Ann E. Woolfrey, B. M. Sandmaier, Monica S. Thakar, Rainer Storb, R Pasquini, Carmen Bonfim, Hans-Peter Kiem, Mark C. Walters |
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| Rok vydání: | 2017 |
| Předmět: |
Male
BMT pediatric medicine.medical_specialty Fanconi’s anemia Cyclophosphamide T-Lymphocytes Clinical Sciences Oncology and Carcinogenesis Immunology Human leukocyte antigen Haploidentical Gastroenterology Article Drug Administration Schedule Lymphocyte Depletion Sepsis 03 medical and health sciences 0302 clinical medicine Fanconi anemia In vivo Internal medicine medicine Humans Preschool Child Transplantation Performance status business.industry haploidentical Hematology medicine.disease 3. Good health Surgery Fanconi Anemia surgical procedures operative Graft-versus-host disease Child Preschool 030220 oncology & carcinogenesis Transplantation Haploidentical Female business Immunosuppressive Agents 030215 immunology medicine.drug |
| Zdroj: | Bone marrow transplantation Bone marrow transplantation, vol 52, iss 4 |
| ISSN: | 1476-5365 0268-3369 |
| Popis: | We developed a haploidentical transplantation protocol with post-transplant cyclophosphamide (CY) for in vivo T-cell depletion using a novel adapted-dosing schedule (25 mg/kg on days +3 and +4) for Fanconi Anemia. With median follow-up of 3 years (range, 37 days to 6.2 years), all six patients engrafted. Two patients with multiple co-morbidities and late referrals to transplant died from sepsis (n=2) and chronic graft-versus-host disease (GVHD) (n=1). Four patients without pre-existing co-morbidities and early transplant referrals are alive with 100% donor chimerism and excellent performance status. We conclude that modulated-dosing post-transplant CY is effective in vivo T-cell depletion to promote full donor engraftment in patients with Fanconi anemia. |
| Databáze: | OpenAIRE |
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