Allogeneic stem cell transplantation is a cure for patients suffering from thalassemia major (TM). Historically, patients were limited by the selection of donors, while the advancement of haploidentical stem cell transplantation (haplo-SCT) has greatly expanded the donor pool. However, the outcomes of haplo-SCT in TM recipients vary between different programs. In this study, we retrospectively studied 73 pediatric TM patients (median age, 7 years; range, 3 to 14 years) who underwent haplo-cord transplantation. Both the estimated overall survival and transfusion-free survival were 95.26% (CI 95.77% to 96.23%). Neither primary nor secondary graft failures were observed. The median follow-up period was 811 days (range, 370 to 1433 days). Median neutrophil and platelet engraftment times were 22 days (range, 8 to 48 days) and 20 days (range, 8 to 99 days), respectively. Acute graft-versus-host disease (aGVHD) was observed in 52% of patients and of these, 25% developed grade III to IV aGVHD. Cord blood engraftment was associated with delayed immune recovery and increased aGVHD severity. Viral DNAemia occurred in a relatively high proportion of patients but only 7% of patients developed CMV disease, while another 7% of patients had post-transplantation lymphoproliferative disorder. Long-term complication outcomes were good. Only one patient developed extensive chronic GVHD. No surviving patients were reliant on blood transfusion by the time this manuscript was submitted. This is one of the largest studies on the outcomes of pediatric TM patients who received stem cell transplantations from alternative donors. The haplo-cord program is safe and practical for TM patients that do not have matched donors.
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Univ Tehran Med Sci, Hematol Oncol & Stem Cell Transplantat Res Ctr, Shariati Hosp, Kargar Shomali Ave, Tehran, IranUniv Tehran Med Sci, Hematol Oncol & Stem Cell Transplantat Res Ctr, Shariati Hosp, Kargar Shomali Ave, Tehran, Iran
Ghavamzadeh, Ardeshir
Sotoudeh, Masoud
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Univ Tehran Med Sci, Digest Dis Res Ctr, Digest Dis Res Inst, Tehran, IranUniv Tehran Med Sci, Hematol Oncol & Stem Cell Transplantat Res Ctr, Shariati Hosp, Kargar Shomali Ave, Tehran, Iran
Sotoudeh, Masoud
Taheri, Amir Pejman Hashemi
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Univ Tehran Med Sci, Dept Radiol, Shariati Hosp, Tehran, IranUniv Tehran Med Sci, Hematol Oncol & Stem Cell Transplantat Res Ctr, Shariati Hosp, Kargar Shomali Ave, Tehran, Iran
Taheri, Amir Pejman Hashemi
Alimoghaddam, Kamran
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Univ Tehran Med Sci, Hematol Oncol & Stem Cell Transplantat Res Ctr, Shariati Hosp, Kargar Shomali Ave, Tehran, IranUniv Tehran Med Sci, Hematol Oncol & Stem Cell Transplantat Res Ctr, Shariati Hosp, Kargar Shomali Ave, Tehran, Iran
Alimoghaddam, Kamran
Pashaiefar, Hossein
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Univ Tehran Med Sci, Dept Med Genet, Sch Med, Tehran, IranUniv Tehran Med Sci, Hematol Oncol & Stem Cell Transplantat Res Ctr, Shariati Hosp, Kargar Shomali Ave, Tehran, Iran
Pashaiefar, Hossein
Jalili, Mahdi
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Univ Tehran Med Sci, Hematol Oncol & Stem Cell Transplantat Res Ctr, Shariati Hosp, Kargar Shomali Ave, Tehran, IranUniv Tehran Med Sci, Hematol Oncol & Stem Cell Transplantat Res Ctr, Shariati Hosp, Kargar Shomali Ave, Tehran, Iran
Jalili, Mahdi
Shahi, Farhad
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Univ Tehran Med Sci, Dept Hematol & Med Oncol, Canc Res Ctr, Canc Inst,Imam Khomeini Hosp Complex, Tehran, IranUniv Tehran Med Sci, Hematol Oncol & Stem Cell Transplantat Res Ctr, Shariati Hosp, Kargar Shomali Ave, Tehran, Iran
Shahi, Farhad
Jahani, Mohammad
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Univ Tehran Med Sci, Hematol Oncol & Stem Cell Transplantat Res Ctr, Shariati Hosp, Kargar Shomali Ave, Tehran, IranUniv Tehran Med Sci, Hematol Oncol & Stem Cell Transplantat Res Ctr, Shariati Hosp, Kargar Shomali Ave, Tehran, Iran
Jahani, Mohammad
Yaghmaie, Marjan
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Univ Tehran Med Sci, Hematol Oncol & Stem Cell Transplantat Res Ctr, Shariati Hosp, Kargar Shomali Ave, Tehran, IranUniv Tehran Med Sci, Hematol Oncol & Stem Cell Transplantat Res Ctr, Shariati Hosp, Kargar Shomali Ave, Tehran, Iran