Hematopoietic stem cell transplantation for homozygous β-thalassemia and β-thalassemia/hemoglobin E patients from haploidentical donors

• Published in: Bone marrow transplantation (Basingstoke) Vol. 51; no. 6; pp. 813 - 818
• Main Authors: Anurathapan, U, Hongeng, S, Pakakasama, S, Sirachainan, N, Songdej, D, Chuansumrit, A, Charoenkwan, P, Jetsrisuparb, A, Sanpakit, K, Rujkijyanont, P, Meekaewkunchorn, A, Lektrakul, Y, Iamsirirak, P, Surapolchai, P, Satayasai, W, Sirireung, S, Sruamsiri, R, Wahidiyat, P A, Ungkanont, A, Issaragrisil, S, Andersson, B S
• Format: Journal Article
• Language: English
• Published: London Nature Publishing Group UK 01-06-2016; Nature Publishing Group
• Subjects: 692/308/2779/109; 692/699/1541/13; Adolescent; Antilymphocyte serum; beta-Thalassemia - therapy; Blood Component Removal; Blood diseases; Busulfan; Cell Biology; Cell survival; Child; Child, Preschool; Chimerism; Cyclophosphamide; Dexamethasone; Disease-Free Survival; Donors; Fludarabine; Globulins; Graft rejection; Graft Survival; Graft-versus-host reaction; Grafts; Hematology; Hematopoietic Stem Cell Transplantation - methods; Hematopoietic Stem Cell Transplantation - mortality; Hematopoietic stem cells; Hemoglobin; Hemoglobin E; Homozygote; Humans; Immunosuppressive agents; Immunosuppressive Agents - therapeutic use; Infant; Internal Medicine; Leukocytes (neutrophilic); Lymphocytes T; Medicine; Medicine & Public Health; Mycophenolate mofetil; Mycophenolic acid; original-article; Peripheral blood; Peripheral Blood Stem Cell Transplantation - methods; Peripheral Blood Stem Cell Transplantation - mortality; Prophylaxis; Public Health; Rapamycin; Stem cell transplantation; Stem Cells; Survival; Survival Rate; Tacrolimus; Thalassemia; Transplantation; Transplantation Conditioning - methods; Transplantation, Haploidentical - methods; Transplantation, Haploidentical - mortality; Young Adult
• ISSN: 0268-3369; 1476-5365
• DOI: 10.1038/bmt.2016.7

Thalassemia-free survival after allogeneic stem cell transplantation (SCT) is about 80–90% with either matched-related or -unrelated donors. We explored the use of a mismatched-related (‘haplo- ’) donor. All patients received two courses of pretransplant immunosuppressive therapy (PTIS) with fludarabine (Flu) and dexamethasone (Dxm). After two courses of PTIS, a conditioning regimen of rabbit antithymocyte globulin, Flu and IV busulfan (Bu) was given followed by T-cell-replete peripheral blood progenitor cells. GvHD prophylaxis consisted of cyclophosphamide (Cy) on days SCT +3 and +4 (post-Cy), and on day SCT +5 tacrolimus or sirolimus was started together with a short course of mycophenolate mofetil. Thirty-one patients underwent haplo-SCT. Their median age was 10 years (range, 2–20 years). Twenty-nine patients engrafted with 100% donor chimerism. Two patients suffered primary graft failure. Median time to neutrophil engraftment was 14 days (range, 11–18 days). Five patients developed mild to moderate, reversible veno-occlusive disease, while nine patients developed acute GvHD grade II. Only five patients developed limited-chronic GvHD. Projected overall and event-free survival rates at 2 years are 95% and 94%, respectively. The median follow up time is 12 months (range, 7–33 months).