Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2023 Jan 24;7(2):227-234.
doi: 10.1182/bloodadvances.2022008137.

Reduction in vaso-occlusive events following stem cell transplantation in patients with sickle cell disease

Alexis Leonard  1   2 , Dana Furstenau  1 , Allistair Abraham  3 , Deepika S Darbari  2 , Robert S Nickel  2 , Emily Limerick  1 , Courtney Fitzhugh  1 , Matt Hsieh  1 , John F Tisdale  1
Affiliations

Reduction in vaso-occlusive events following stem cell transplantation in patients with sickle cell disease

Alexis Leonard et al. Blood Adv. .

Abstract

Hematopoietic stem cell transplantation (HSCT) is potentially curative for patients with sickle cell disease (SCD). Patients with stable donor engraftment after allogeneic HSCT generally do not experience SCD-related complications; however, there are no published data specifically reporting the change in vaso-occlusive events (VOE) after HSCT. Data regarding the number of VOEs requiring medical attention in the 2 years before allogeneic HSCT were compared with the number of VOEs in the 2 years (0-12 months and 12-24 months) after allogeneic HSCT in patients with SCD. One-hundred sixty-three patients with SCD underwent allogeneic HSCT between 2005 and 2019. The average age at the time of HSCT was 21 years (range, 7 months - 64 years). Most patients underwent nonmyeloablative conditioning (75% [N = 123]) and had a matched sibling donor (72% [N = 118]). The mean number of VOEs was reduced from 5.6 (range, 0-52) in the 2 years before HSCT to 0.9 (range, 0-12) in the 2 years after HSCT (P < .001). Among the post-HSCT events, VOE was more frequent during the first 12 months (0.8 [range, 0-12]) than at 12 to 24 months after HSCT (0.1 [range, 0-8) (P < .001)). In patients who had graft rejection (12%, N = 20), VOEs were reduced from 6.6 (range, 0-24) before HSCT to 1.1 (range, 0-6) and 0.8 (range, 0-8) at 0 to 12 months and 12 to 24 months after HSCT, respectively (P < .001). VOEs requiring medical care were significantly reduced after allogeneic HSCT for patients with SCD. These data will inform the development of novel autologous HSCT gene therapy approaches.

Trial registration: ClinicalTrials.gov NCT00061568 NCT02105766 NCT03077542 NCT00977691 NCT02165007 NCT03587272 NCT00745420 NCT02766465 NCT03263559 NCT04018937 NCT00029380.

PubMed Disclaimer

Conflict of interest statement

Conflict-of-interest disclosure: A.A. served on the safety monitoring committee for Sangamo Therapeutics. The remaining authors declare no competing financial interests.

Figures

None
Graphical abstract
Figure 1.
Figure 1.
Number of VOEs before and after HSCT. HSCT, hematopoietic stem cell transplantation; GVHD, graft-vs-host disease; VOE, vaso-occlusive event.
Figure 2.
Figure 2.
Number of VOEs before and after HSCT in engrafted patients with and without GVHD and in patients with graft rejection. GVHD, graft-vs-host disease; PRE, pretransplantation; POST, post-transplantation; VOE, vaso-occlusive event.
Figure 3.
Figure 3.
Number of VOEs before and after HSCT in patients with graft rejection. Number of VOEs before and after transplant in 20 patients with graft rejection displayed by (a) sickle hemoglobin and (b) fetal hemoglobin percentages at 2 years post transplantation. (C) Fetal hemoglobin percentages at the time of transplant vs 2 years post transplantation. HbF, fetal hemoglobin; HbS, sickle hemoglobin; HSCT, hematopoietic stem cell transplantation; VOE, vaso-occlusive event.
See this image and copyright information in PMC

References

    1. Paulukonis ST, Eckman JR, Snyder AB, et al. Defining sickle cell disease mortality using a population-based surveillance system, 2004 through 2008. Public Health Rep. 2016;131(2):367–375. - PMC - PubMed
    1. Lanzkron S, Carroll CP, Haywood C., Jr. Mortality rates and age at death from sickle cell disease: U.S., 1979-2005. Public Health Rep. 2013;128(2):110–116. - PMC - PubMed
    1. Shenoy S, Angelucci E, Arnold SD, et al. 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. Biol Blood Marrow Transplant. 2017;23(4):552–561. - PubMed
    1. Shenoy S, Gaziev J, Angelucci E, et al. Late effects screening guidelines after hematopoietic cell transplantation (HCT) for hemoglobinopathy: consensus statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on late effects after pediatric HCT. Biol Blood Marrow Transplant. 2018;24(7):1313–1321. - PubMed
    1. Darbari DS, Liljencrantz J, Ikechi A, et al. Pain and opioid use after reversal of sickle cell disease following HLA-matched sibling haematopoietic stem cell transplant. Br J Haematol. 2019;184(4):690–693. - PMC - PubMed

Publication types

MeSH terms

Associated data