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Biol Blood Marrow Transplant. 2017 Apr;23(4):552-561. doi: 10.1016/j.bbmt.2017.01.009. Epub 2017 Jan 5.

Current Results and Future Research Priorities in Late Effects after Hematopoietic Stem Cell Transplantation for Children with Sickle Cell Disease and Thalassemia: A Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric Hematopoietic Stem Cell Transplantation.

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Department of Pediatrics, Washington University School of Medicine and St. Louis Children's Hospital, St. Louis, Missouri. Electronic address:
Department of Hematology, Ospedale Oncologico di Riferimento Regionale "Armando Businco", Cagliari, Italy; Department of Hematology, IRCCS Azienda Ospedaliera Universitaria San Martino-IST Istituto Nazionale per la Ricerca sul Cancro, Genova, Italy.
Department of Pediatrics, Emory University School of Medicine, Atlanta, Georgia.
Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington.
Department of Pediatrics, Columbia University Medical Center, New York, New York.
Willem-Alexander Children's Hospital, LUMC, Leiden, The Netherlands.
Division of Hematology, Oncology, and BMT, Children's Hospital Los Angeles, Los Angeles, California.
Department of Pediatrics, Imperial College Healthcare, London, United Kingdom.
Department of Pediatrics, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, Massachusetts.
International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinico Tor Vergata, Rome, Italy.
Department of Pediatrics, Washington University School of Medicine and St. Louis Children's Hospital, St. Louis, Missouri; Program in Occupational Therapy, Division of Public Health Sciences, Department of Surgery, Washington University School of Medicine, St. Louis, Missouri.
Department of Pediatrics, University of Minnesota Children's Hospital, Minneapolis, Minnesota.
Department of Pediatrics, UCSF Benioff Children's Hospital, Oakland, California.


Sustained donor engraftment after allogeneic hematopoietic cell transplantation (HCT) converts to healthy donor hemoglobin synthesis and halts disease symptoms in patients with sickle cell disease and thalassemia major. A disease-free survival probability that exceeds 90% has been reported when HCT using an HLA-matched sibling donor is performed in young patients with low-risk disease or treatment-related risk factors. Alternate donor HCT and HCT in adults is performed infrequently because of a higher risk profile. Transplant-specific risks include conditioning regimen-related toxicity, graft-versus-host disease, graft rejection with marrow aplasia or disease recurrence, and infections associated with immunosuppression and delayed immune reconstitution. The magnitude of risk depends on patient age, clinical status of the underlying disease (eg, organ injury from vasculopathy and iron overload), donor source, and intensity of the conditioning regimen. These risks are commonly monitored and reported in the short term. Documenting very late outcomes is important, but these data are rarely reported because of challenges imposed by patient drop-out and insufficient resources. This report summarizes long-term follow-up results after HCT for hemoglobin disorders, identifies gaps in knowledge, and discusses opportunities for future investigations. This consensus summary will be followed by a second article detailing comprehensive long-term follow-up recommendations to aid in maintaining health in these individuals and identifying late complication risks that could facilitate interventions to improve outcomes.


Late effects; Pediatric stem cell transplant; Sickle cell disease; Thalassemia

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