Long-Term Follow-Up after Reduced-Intensity Conditioning and Stem Cell Transplantation for Childhood Nonmalignant Disorders

Lisa M. Madden, Robert J. Hayashi, Ka Wah Chan, Michael A. Pulsipher, Dorothea Douglas, Gregory A. Hale, Sonali Chaudhury, Paul Haut, Kimberly A. Kasow, Andrew L. Gilman, Lisa M. Murray, Shalini Shenoy

Research output: Contribution to journalArticle

24 Scopus citations


Reduced-intensity conditioning (RIC) before hematopoietic stem cell transplantation (HCT) in children could result in fewer complications during follow-up compared with myeloablative regimens. Hence, many RIC regimens are under investigation, but long-term follow-up is essential. We describe late follow-up beyond 2 years post-HCT in 43 children with nonmalignant disorders who underwent related or unrelated donor (56%) HCT on a multicenter study using a RIC regimen (alemtuzumab, fludarabine, and melphalan) followed by bone marrow (n = 30), peripheral blood (n = 3), or umbilical cord blood (n = 10) HCT for immune dysfunction, bone marrow failure, metabolic disorders, or hemoglobinopathy. Recipients (median age, 7.5 years; range, 3 to 26) underwent HCT 2 to 8 years (median, 3.1 years) before this report. Full donor (67%) or stable mixed chimerism (33%) was noted without late graft rejection. Five patients (12%) required systemic immunosuppression therapy (IST) beyond 2 years post-HCT for graft-versus-host disease (GVHD); 2 patients died 38 and 79 months later, whereas the others improved, enabling an IST wean. Overall, 17 complications were documented in 10 patients (23%). Complications not related to GVHD included hypothyroidism (n = 2), low grade neoplasms (n = 2), and delayed puberty (n = 1). One patient with GVHD had ovarian failure; all other postpubertal females resumed normal ovarian function. Twenty-seven of 28 school-age recipients were functioning at grade level. RIC HCT recipients thus had few regimen-related toxicities during long-term follow-up. However, objective long-term follow-up is still necessary to identify complications so timely intervention may be planned.

Original languageEnglish (US)
Pages (from-to)1467-1472
Number of pages6
JournalBiology of Blood and Marrow Transplantation
Issue number8
StatePublished - Aug 1 2016



  • Childhood nonmalignant disorders
  • Late complications
  • Reduced-intensity conditioning
  • Stem cell transplantation

ASJC Scopus subject areas

  • Medicine(all)
  • Hematology
  • Transplantation

Cite this

Madden, L. M., Hayashi, R. J., Chan, K. W., Pulsipher, M. A., Douglas, D., Hale, G. A., Chaudhury, S., Haut, P., Kasow, K. A., Gilman, A. L., Murray, L. M., & Shenoy, S. (2016). Long-Term Follow-Up after Reduced-Intensity Conditioning and Stem Cell Transplantation for Childhood Nonmalignant Disorders. Biology of Blood and Marrow Transplantation, 22(8), 1467-1472. https://doi.org/10.1016/j.bbmt.2016.04.025