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Systematic Review Meta-Analysis Journal Article Ppublish 7 citations

PubMed: 38517255 DOI: 10.1002/ajh.27297

Effect of allogeneic hematopoietic stem cell transplantation on sickle cell disease-related organ complications: A systematic review and meta-analysis.

Am J Hematol 99(6):1129-1141

Authors: Elisabeth Dovern, Mesire Aydin, Michael R DeBaun, Komeil Alizade, Bart J Biemond, Erfan Nur

Affiliations

  • Department of Hematology, Amsterdam University Medical Centers, location University of Amsterdam, Amsterdam, The Netherlands.
  • Department of Pediatrics, Vanderbilt-Meharry Center of Excellence in Sickle Cell Disease, Vanderbilt University Medical Center, Nashville, USA.
  • Department of Blood Cell Research, Sanquin Research, Amsterdam, The Netherlands.

Abstract

Sickle cell disease (SCD)-related organ complications are a major cause of morbidity and mortality in patients with SCD. We sought to assess whether hematopoietic stem cell transplantation (HSCT) stabilizes, attenuates, or exacerbates organ decline. We performed a systematic review and meta-analysis of trials investigating organ function before and after HSCT in patients with SCD. We searched MEDLINE/PubMed and EMBASE up to September 21, 2023. Continuous data were expressed as standardized mean difference (SMD) and pooled in a weighted inverse-variance random-effects model; binomial data were expressed as risk ratio (RR) using the Mantel-Haenszel random-effects meta-analyses. Of 823 screened studies, 34 were included in this review. Of these, 17 (774 patients, 23.6% adults, 86.3% HLA-identical sibling donor, 56.7% myeloablative conditioning regimen) were included in the meta-analyses. Pulmonary function remained stable. Mean tricuspid regurgitant jet velocity decreased but did not reach statistical significance. In children, estimated glomerular filtration rate decreased (SMD -0.80, p = .01), and the presence of proteinuria increased (RR 2.00, p = <.01), while splenic uptake and phagocytic function improved (RR 0.31, p = <.01; RR 0.23, p = <.01). Cerebral blood flow improved (SMD -1.39, p = <.01), and a low incidence of stroke after transplantation in high-risk patients was found. Retinopathy and avascular osteonecrosis were investigated in only one study, showing no significant changes. While HSCT can improve some SCD-related organ dysfunctions, transplantation-related toxicity may have an adverse effect on others. Future research should focus on identifying individuals with SCD who might benefit most from HSCT and which forms of organ damage are more likely to exacerbate post-transplantation.

MeSH

Cells, Hematology, Microscopy, Electron, Scanning Transmission, Anemia, Sickle Cell, Hematopoietic Stem Cell Transplantation, Stem Cells, Cell Transplantation, Stem Cell Transplantation, Hematopoietic Stem Cells, Transplantation

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