Late Effects of Hemopoietic Stem Cell Transplant for Sickle Cell Disease: Monitoring and Management

Overview

Journal Expert Rev Hematol

Specialty Hematology

Date 2024 Nov 5

PMID 39499235

Authors

Marti Goldenberg

Sophie Lanzkron

Lydia H Pecker

Affiliations

Soon will be listed here.

Abstract

Introduction: Allogeneic hemopoietic stem cell transplantation (HSCT) is a curative therapy for sickle cell disease (SCD). Exposure to both SCD and HSCT conditioning regimens is associated with late health effects.

Areas Covered: This review addresses post-HSCT outcomes and late health effects among individuals with SCD exposed to allogeneic HSCT regimens, summarizes recommendations for long-term care, and identifies future survivorship research needs.

Expert Opinion: Individuals with SCD exposed to HSCT and gene therapy require multidisciplinary care to monitor late health effects. To optimize care, multi-disciplinary clinics that include experts in late effects of HSCT exposure, SCD, complex chronic pain, mental health, and social work are needed. Research defining the late effects of exposure is needed to inform patient management and build clinical care infrastructure.

References

Hsieh M, Kang E, Fitzhugh C, Link M, Bolan C, Kurlander R . Allogeneic hematopoietic stem-cell transplantation for sickle cell disease. N Engl J Med. 2009; 361(24):2309-17. PMC: 3627532. DOI: 10.1056/NEJMoa0904971. View

Gladwin M, Sachdev V, Jison M, Shizukuda Y, Plehn J, Minter K . Pulmonary hypertension as a risk factor for death in patients with sickle cell disease. N Engl J Med. 2004; 350(9):886-95. DOI: 10.1056/NEJMoa035477. View

Schoettler M, Stenger E, Spencer K, Lutterman D, Rumbika S, Jones J . Sickle cell disease is a risk factor for transplant-associated thrombotic microangiopathy in children. Blood Adv. 2022; 7(9):1784-1795. PMC: 10164784. DOI: 10.1182/bloodadvances.2022008058. View

Fitzhugh C, Hsieh M, Taylor T, Coles W, Roskom K, Wilson D . Cyclophosphamide improves engraftment in patients with SCD and severe organ damage who undergo haploidentical PBSCT. Blood Adv. 2018; 1(11):652-661. PMC: 5727815. DOI: 10.1182/bloodadvances.2016002972. View

Brunson A, Keegan T, Bang H, Mahajan A, Paulukonis S, Wun T . Increased risk of leukemia among sickle cell disease patients in California. Blood. 2017; 130(13):1597-1599. PMC: 5620417. DOI: 10.1182/blood-2017-05-783233. View

Chakravarthy R, Friedman D . Long-term health outcomes following curative therapies for sickle cell disease. Hematology Am Soc Hematol Educ Program. 2022; 2022(1):272-276. PMC: 9820909. DOI: 10.1182/hematology.2022000373. View

Pecker L, Oteng-Ntim E, Nero A, Lanzkron S, Christianson M, Woolford T . Expecting more: the case for incorporating fertility services into comprehensive sickle cell disease care. Lancet Haematol. 2023; 10(3):e225-e234. PMC: 10318482. DOI: 10.1016/S2352-3026(22)00353-2. View

Azevedo J, Malmegrim K . Immune mechanisms involved in sickle cell disease pathogenesis: current knowledge and perspectives. Immunol Lett. 2020; 224:1-11. DOI: 10.1016/j.imlet.2020.04.012. View

Pecker L, Akinsete A, Carroll C, Lanzkron S, Kuo K, Hulbert M . Life after sickle cell disease, is it really uhuru?. Lancet Haematol. 2023; 10(12):e946-e949. PMC: 10957145. DOI: 10.1016/S2352-3026(23)00306-X. View

10.

Krishnamurti L . Hematopoietic Cell Transplantation for Sickle Cell Disease. Front Pediatr. 2021; 8:551170. PMC: 7813811. DOI: 10.3389/fped.2020.551170. View

11.

Hasan S, Hashmi S, Alhassen M, Lawson W, Castro O . Depression in sickle cell disease. J Natl Med Assoc. 2003; 95(7):533-7. PMC: 2594635. View

12.

Walters M, De Castro L, Sullivan K, Krishnamurti L, Kamani N, Bredeson C . Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease. Biol Blood Marrow Transplant. 2015; 22(2):207-211. PMC: 5031360. DOI: 10.1016/j.bbmt.2015.10.017. View

13.

Pedersen S, Monagel D, Mammen C, Lewis V, Guilcher G, Bruce A . Stable renal function in children and adolescents with sickle cell disease after nonmyeloablative hematopoietic stem cell transplantation. Pediatr Blood Cancer. 2020; 67(9):e28568. DOI: 10.1002/pbc.28568. View

14.

Shenoy S, Angelucci E, Arnold S, Baker K, Bhatia M, Bresters D . 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.... Biol Blood Marrow Transplant. 2017; 23(4):552-561. DOI: 10.1016/j.bbmt.2017.01.009. View

15.

Pearson H, Cornelius E, Schwartz A, Zelson J, WOLFSON S, Spencer R . Transfusion-reversible functional asplenia in young children with sickle-cell anemia. N Engl J Med. 1970; 283(7):334-7. DOI: 10.1056/NEJM197008132830703. View

16.

Allen E, Srivastava K, Hsieh M, Fitzhugh C, Klein H, Tisdale J . Immunohaematological complications in patients with sickle cell disease after haemopoietic progenitor cell transplantation: a prospective, single-centre, observational study. Lancet Haematol. 2017; 4(11):e553-e561. PMC: 6311987. DOI: 10.1016/S2352-3026(17)30196-5. View

17.

Saraf S, Oh A, Patel P, Jalundhwala Y, Sweiss K, Koshy M . Nonmyeloablative Stem Cell Transplantation with Alemtuzumab/Low-Dose Irradiation to Cure and Improve the Quality of Life of Adults with Sickle Cell Disease. Biol Blood Marrow Transplant. 2015; 22(3):441-8. DOI: 10.1016/j.bbmt.2015.08.036. View

18.

Fitzhugh C . Knowledge to date on secondary malignancy following hematopoietic cell transplantation for sickle cell disease. Hematology Am Soc Hematol Educ Program. 2022; 2022(1):266-271. PMC: 9820448. DOI: 10.1182/hematology.2022000371. View

19.

Locatelli F, Rocha V, Reed W, Bernaudin F, Ertem M, Grafakos S . Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease. Blood. 2002; 101(6):2137-43. DOI: 10.1182/blood-2002-07-2090. View

20.

Majhail N, Rizzo J, Lee S, Aljurf M, Atsuta Y, Bonfim C . Recommended screening and preventive practices for long-term survivors after hematopoietic cell transplantation. Biol Blood Marrow Transplant. 2011; 18(3):348-71. PMC: 3279570. DOI: 10.1016/j.bbmt.2011.12.519. View