Outcome of hematopoietic cell transplantation in children with sickle cell disease, a single center's experience

S. Majumdar, Z. Robertson, A. Robinson, S. Starnes, R. Iyer, G. Megason

Research output: Contribution to journalArticle

24 Citations (Scopus)

Abstract

Multicenter trials have shown that hematopoietic cell transplantation (HCT) has an excellent outcome in children with sickle cell disease (SCD). As a single center, we performed a total of 11 transplants in 10 patients (6 males, 4 females) with SCD between 1997 and 2005. Eight patients had hemoglobin SS disease and two patients had HbSΒ 0 thalassemia. The median age of HCT was 10.1 (range 2.8-16.3) years. All donors were HLA-identical siblings; six patients received bone marrow (BM), two patients received mobilized peripheral blood, one patient received umbilical cord blood (UCB) and one patient received both UCB and BM from the same donor. Myeloablative conditioning regimen consisted of busulfan, horse antithymocyte globulin and cyclophosphamide. One patient had a gradual decrease in donor chimerism to 15% and subsequently received a second bone marrow transplant using a reduced intensity conditioning regimen consisting of alemtuzumab, fludarabine and melphalan leading to stable full engraftment. Currently, 9 out of 10 patients are alive with a median follow-up of 5.5 (range 2.9-11) years. As a single institution's experience with HCT in children with SCD, we report an excellent outcome, and a second HCT may be considered for patients with impending engraftment failure as a cure for SCD.

Original languageEnglish (US)
Pages (from-to)895-900
Number of pages6
JournalBone Marrow Transplantation
Volume45
Issue number5
DOIs
StatePublished - May 2010

Fingerprint

Cell Transplantation
Sickle Cell Anemia
Bone Marrow
Tissue Donors
Fetal Blood
Transplants
Sickle Hemoglobin
Busulfan
Chimerism
Melphalan
Antilymphocyte Serum
Thalassemia
Cyclophosphamide
Horses
Multicenter Studies
Siblings

Keywords

  • Hematopoietic stem cell transplantation
  • Long-term outcome
  • Sickle cell disease

ASJC Scopus subject areas

  • Hematology
  • Transplantation

Cite this

Outcome of hematopoietic cell transplantation in children with sickle cell disease, a single center's experience. / Majumdar, S.; Robertson, Z.; Robinson, A.; Starnes, S.; Iyer, R.; Megason, G.

In: Bone Marrow Transplantation, Vol. 45, No. 5, 05.2010, p. 895-900.

Research output: Contribution to journalArticle

Majumdar, S. ; Robertson, Z. ; Robinson, A. ; Starnes, S. ; Iyer, R. ; Megason, G. / Outcome of hematopoietic cell transplantation in children with sickle cell disease, a single center's experience. In: Bone Marrow Transplantation. 2010 ; Vol. 45, No. 5. pp. 895-900.
@article{6e725213d5f942a7a3532d77d1b1fa2f,
title = "Outcome of hematopoietic cell transplantation in children with sickle cell disease, a single center's experience",
abstract = "Multicenter trials have shown that hematopoietic cell transplantation (HCT) has an excellent outcome in children with sickle cell disease (SCD). As a single center, we performed a total of 11 transplants in 10 patients (6 males, 4 females) with SCD between 1997 and 2005. Eight patients had hemoglobin SS disease and two patients had HbSΒ 0 thalassemia. The median age of HCT was 10.1 (range 2.8-16.3) years. All donors were HLA-identical siblings; six patients received bone marrow (BM), two patients received mobilized peripheral blood, one patient received umbilical cord blood (UCB) and one patient received both UCB and BM from the same donor. Myeloablative conditioning regimen consisted of busulfan, horse antithymocyte globulin and cyclophosphamide. One patient had a gradual decrease in donor chimerism to 15{\%} and subsequently received a second bone marrow transplant using a reduced intensity conditioning regimen consisting of alemtuzumab, fludarabine and melphalan leading to stable full engraftment. Currently, 9 out of 10 patients are alive with a median follow-up of 5.5 (range 2.9-11) years. As a single institution's experience with HCT in children with SCD, we report an excellent outcome, and a second HCT may be considered for patients with impending engraftment failure as a cure for SCD.",
keywords = "Hematopoietic stem cell transplantation, Long-term outcome, Sickle cell disease",
author = "S. Majumdar and Z. Robertson and A. Robinson and S. Starnes and R. Iyer and G. Megason",
year = "2010",
month = "5",
doi = "10.1038/bmt.2009.244",
language = "English (US)",
volume = "45",
pages = "895--900",
journal = "Bone Marrow Transplantation",
issn = "0268-3369",
publisher = "Nature Publishing Group",
number = "5",

}

TY - JOUR

T1 - Outcome of hematopoietic cell transplantation in children with sickle cell disease, a single center's experience

AU - Majumdar, S.

AU - Robertson, Z.

AU - Robinson, A.

AU - Starnes, S.

AU - Iyer, R.

AU - Megason, G.

PY - 2010/5

Y1 - 2010/5

N2 - Multicenter trials have shown that hematopoietic cell transplantation (HCT) has an excellent outcome in children with sickle cell disease (SCD). As a single center, we performed a total of 11 transplants in 10 patients (6 males, 4 females) with SCD between 1997 and 2005. Eight patients had hemoglobin SS disease and two patients had HbSΒ 0 thalassemia. The median age of HCT was 10.1 (range 2.8-16.3) years. All donors were HLA-identical siblings; six patients received bone marrow (BM), two patients received mobilized peripheral blood, one patient received umbilical cord blood (UCB) and one patient received both UCB and BM from the same donor. Myeloablative conditioning regimen consisted of busulfan, horse antithymocyte globulin and cyclophosphamide. One patient had a gradual decrease in donor chimerism to 15% and subsequently received a second bone marrow transplant using a reduced intensity conditioning regimen consisting of alemtuzumab, fludarabine and melphalan leading to stable full engraftment. Currently, 9 out of 10 patients are alive with a median follow-up of 5.5 (range 2.9-11) years. As a single institution's experience with HCT in children with SCD, we report an excellent outcome, and a second HCT may be considered for patients with impending engraftment failure as a cure for SCD.

AB - Multicenter trials have shown that hematopoietic cell transplantation (HCT) has an excellent outcome in children with sickle cell disease (SCD). As a single center, we performed a total of 11 transplants in 10 patients (6 males, 4 females) with SCD between 1997 and 2005. Eight patients had hemoglobin SS disease and two patients had HbSΒ 0 thalassemia. The median age of HCT was 10.1 (range 2.8-16.3) years. All donors were HLA-identical siblings; six patients received bone marrow (BM), two patients received mobilized peripheral blood, one patient received umbilical cord blood (UCB) and one patient received both UCB and BM from the same donor. Myeloablative conditioning regimen consisted of busulfan, horse antithymocyte globulin and cyclophosphamide. One patient had a gradual decrease in donor chimerism to 15% and subsequently received a second bone marrow transplant using a reduced intensity conditioning regimen consisting of alemtuzumab, fludarabine and melphalan leading to stable full engraftment. Currently, 9 out of 10 patients are alive with a median follow-up of 5.5 (range 2.9-11) years. As a single institution's experience with HCT in children with SCD, we report an excellent outcome, and a second HCT may be considered for patients with impending engraftment failure as a cure for SCD.

KW - Hematopoietic stem cell transplantation

KW - Long-term outcome

KW - Sickle cell disease

UR - http://www.scopus.com/inward/record.url?scp=77952427892&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=77952427892&partnerID=8YFLogxK

U2 - 10.1038/bmt.2009.244

DO - 10.1038/bmt.2009.244

M3 - Article

C2 - 19784079

AN - SCOPUS:77952427892

VL - 45

SP - 895

EP - 900

JO - Bone Marrow Transplantation

JF - Bone Marrow Transplantation

SN - 0268-3369

IS - 5

ER -