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Inherited and Genetic Disorders

Long-term outcomes of fludarabine, melphalan and antithymocyte globulin as reduced-intensity conditioning regimen for allogeneic hematopoietic stem cell transplantation in children with primary immunodeficiency disorders: a prospective single center study

Bone Marrow Transplantation volume 51, pages 219–226 (2016)Cite this article

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Abstract

Reduced-intensity conditioning (RIC) has offered many primary immunodeficiency disorder (PID) patients who are ineligible for myeloablative regimens a chance of cure. However, the beneficial role of RIC was questioned following reports suggesting higher chance of rejection and lower symptom resolution rate in mixed chimerism settings. Forty-five children affected by PIDs with a median age of 21 months underwent allogeneic hematopoietic stem cell transplantation in our institute from 2007 to 2013. All patients received an identical RIC regimen. Forty-one patients had successful primary engraftment (91%). Of the successful engraftments, 80% (n=33) had stable full donor chimerism at last contact. Overall, eleven transplant-related mortalities were reported including five patients due to sepsis, three children due to grade IV acute GvHD, two due to chronic GvHD and one patient due to sepsis after primary graft failure. The median post-transplantation follow-up of deceased patients was 55 days. Five-year overall survival and disease-free survival was 75.6% and 68.89%, respectively. All surviving patients with successful engraftment became disease free, regardless of having full or mixed chimerism. Our study suggests that RIC regimen provides satisfactory rates of successful engraftment and full chimerism. Furthermore, patients with mixed chimerism were stable in long-term follow-up and this chimerism status offered the potential to resolve symptoms of immunodeficiency.

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Acknowledgements

We are grateful for physicians’ help in diagnosing and referring patients. We sincerely thank all patients and their families for their participation in this study and the medical, nursing and clinical staff for their devoted care of patients.

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Authors and Affiliations

  1. Hematology, Oncology and Stem Cell Transplantation Research Centre, Tehran University of Medical Sciences, Tehran, Iran

    A A Hamidieh, M Behfar, S Faghihi-Kashani, A S Hosseini, M Mozafari & A Ghavamzadeh

  2. Immunology, Asthma and Allergy Research Institute, Tehran University of Medical Sciences, Tehran, Iran

    Z Pourpak, M R Fazlollahi, M Movahedi & M Moin

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  1. A A Hamidieh
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Correspondence to A A Hamidieh.

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Hamidieh, A., Behfar, M., Pourpak, Z. et al. Long-term outcomes of fludarabine, melphalan and antithymocyte globulin as reduced-intensity conditioning regimen for allogeneic hematopoietic stem cell transplantation in children with primary immunodeficiency disorders: a prospective single center study. Bone Marrow Transplant 51, 219–226 (2016). https://doi.org/10.1038/bmt.2015.277

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