Long-term outcomes after unrelated donor transplantation for severe sickle cell disease on the BMT CTN 0601 trial

Mary Eapen¹, Jianqun Kou², Martin Andreansky^{3

4}, Monica Bhatia⁵, Joel Brochstein⁶, Sonali Chaudhury⁷, Ann E Haight⁸, Hilary Haines⁹, David Jacobsohn¹⁰, Jennifer Jaroscak¹¹, Kimberly A Kasow¹², Lakshmanan Krishnamurti^{13

14}, John E Levine^{15

16}, Kathryn Leung^{8

17}, David Margolis¹⁸, Lolie C Yu¹⁹, Mary M Horowitz^{1

2}, Naynesh Kamani¹⁰, Mark C Walters²⁰, Shalini Shenoy²¹

Affiliations

¹ Department of Medicine, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.
² Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.
³ Department of Pediatrics, University of Miami, Miami, Florida, USA.
⁴ Christus Children's Hospital, Baylor College of Medicine, San Antonio, Texas, USA.
⁵ Department of Pediatrics, Columbia University Medical Center, New York, New York, USA.
⁶ Department of Pediatrics, Cohen Children's Medical Center, New Hyde Park, New York, USA.
⁷ Department of Pediatrics, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.
⁸ Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA.
⁹ Department of Pediatrics, University of Alabama, Birmingham, Alabama, USA.
¹⁰ Department of Pediatrics, Children's National Hospital, Washington, DC, USA.
¹¹ Department of Pediatrics, Medical University of South Carolina, Charleston, South Carolina, USA.
¹² Department of Pediatrics, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA.
¹³ Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, USA.
¹⁴ Department of Pediatrics, Yale School of Medicine, New Haven, Connecticut, USA.
¹⁵ Department of Internal Medicine and Pediatrics, University of Michigan, Ann Arbor, Michigan, USA.
¹⁶ Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, New York, USA.
¹⁷ Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA.
¹⁸ Department of Pediatrics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.
¹⁹ Louisiana State University Medical Center, Children's Hospital of New Orleans, New Orleans, Louisiana, USA.
²⁰ Department of Pediatrics, UCSF Benioff Children's Hospitals, University of California San Francisco, San Francisco, California, USA.
²¹ Department of Pediatrics, Washington University School of Medicine, St. Louis Children's Hospital, St. Louis, Missouri, USA.

PMID: 38343182
PMCID: PMC10947844
DOI: 10.1002/ajh.27251

Long-term outcomes after unrelated donor transplantation for severe sickle cell disease on the BMT CTN 0601 trial

Mary Eapen et al. Am J Hematol. 2024 Apr.

. 2024 Apr;99(4):785-788.

doi: 10.1002/ajh.27251. Epub 2024 Feb 11.

Authors

Affiliations

¹ Department of Medicine, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.
² Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.
³ Department of Pediatrics, University of Miami, Miami, Florida, USA.
⁴ Christus Children's Hospital, Baylor College of Medicine, San Antonio, Texas, USA.
⁵ Department of Pediatrics, Columbia University Medical Center, New York, New York, USA.
⁶ Department of Pediatrics, Cohen Children's Medical Center, New Hyde Park, New York, USA.
⁷ Department of Pediatrics, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.
⁸ Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA.
⁹ Department of Pediatrics, University of Alabama, Birmingham, Alabama, USA.
¹⁰ Department of Pediatrics, Children's National Hospital, Washington, DC, USA.
¹¹ Department of Pediatrics, Medical University of South Carolina, Charleston, South Carolina, USA.
¹² Department of Pediatrics, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA.
¹³ Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, USA.
¹⁴ Department of Pediatrics, Yale School of Medicine, New Haven, Connecticut, USA.
¹⁵ Department of Internal Medicine and Pediatrics, University of Michigan, Ann Arbor, Michigan, USA.
¹⁶ Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, New York, USA.
¹⁷ Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA.
¹⁸ Department of Pediatrics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.
¹⁹ Louisiana State University Medical Center, Children's Hospital of New Orleans, New Orleans, Louisiana, USA.
²⁰ Department of Pediatrics, UCSF Benioff Children's Hospitals, University of California San Francisco, San Francisco, California, USA.
²¹ Department of Pediatrics, Washington University School of Medicine, St. Louis Children's Hospital, St. Louis, Missouri, USA.

PMID: 38343182
PMCID: PMC10947844
DOI: 10.1002/ajh.27251

No abstract available

PubMed Disclaimer

Conflict of interest statement

CONFLICT OF INTEREST

Authors declare none for this study

Figures

**Figure 1**
Overall Survival and Event-Free survival

See this image and copyright information in PMC

References

1. Shenoy S, Eapen M, Panepinto JA, et al. A trial of unrelated donor marrow transplantation for children with severe sickle cell disease. Blood. 2016; 128(21): 2561 – 2567 - PMC - PubMed
1. Gluckman E, Cappelli B, Scigliuolo GM, De la Fuente J, Corbacioglu S. Alternative donor hematopoietic stem cell transplantation for sickle cell disease in Europe. Hematol Oncol Stem Cell Ther. 2020;13(4):181–188. - PubMed
1. Smith-Whitley K Reproductive issues in sickle cell disease. Hematology Am Soc Hematol Educ Program. 2014;2014(1):418–24. - PubMed
1. Kinger NP, Moreno CC, Miller FH, Mittal PK. Abdominal Manifestations of Sickle Cell Disease. Curr Probl Diagn Radiol. 2021;50(2):241–251. - PubMed
1. Pecker LH, Darbari DS. Psychosocial and affective comorbidities in sickle cell disease. Neurosci Lett. 2019;705:1–6. - PubMed

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Long-term outcomes after unrelated donor transplantation for severe sickle cell disease on the BMT CTN 0601 trial

Affiliations

Long-term outcomes after unrelated donor transplantation for severe sickle cell disease on the BMT CTN 0601 trial

Authors

Affiliations

Conflict of interest statement

Figures

References

Publication types

MeSH terms

Grants and funding

LinkOut - more resources

Full Text Sources

Medical