Details

Autor(en) / Beteiligte

• 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

Titel

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

Ist Teil von

• Bone marrow transplantation (Basingstoke), 2016-06, Vol.51 (6), p.813-818

Ort / Verlag

England: Nature Publishing Group

Erscheinungsjahr

2016

Quelle

MEDLINE

Beschreibungen/Notizen

• 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).