ArticleNeurology2026
Hematopoietic Stem Cell Transplant and Brain Volume Changes in Adults With Sickle Cell Disease.
Article in Neurology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 5 registered trials, which are not on this map. Not yet cited in PubMed.
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Nonmyeloablative Allogeneic Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Severe Congenital Anemias Including Sickle Cell Disease (SCD) and B-Thalassemia
Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Severe Congenital Anemias Including Sickle Cell Disease and Beta-Thalassemia
Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia in Individuals With Higher Risk of Transplant Failure
A Phase 1/2 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With the LentiGlobin BB305 Lentiviral Vector in Subjects With Severe Sickle Cell Disease
Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease
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Authors and funding
10 authors.
Funding
Abstract
BACKGROUND AND
objectivesAdults with sickle cell disease (SCD) are at risk of decline in brain health and cognition, even without clinical stroke. SCD-related changes on MRI include brain atrophy and T2 fluid attenuated inversion recovery (FLAIR) white matter hyperintensity (WMH) growth. Hematopoietic stem cell transplant (HSCT) can be curative, but conditioning regimens risk new complications, particularly for adults with accumulated organ damage. We tested whether successful HSCT for adults with SCD was associated with reduced brain atrophy or WMH growth.
methodsIn this cohort study, adults with severe SCD underwent HSCT as part of 5 research protocols at the National Institutes of Health Clinical Center, including autologous gene therapy, HLA-matched donor HSCs, and haploidentical HSCs. Serial brain MRI scans acquired before and after transplant for up to 12 years included 3D T1-weighted and axial FLAIR sequences. Longitudinal brain volumes, segmented using FreeSurfer and corrected for total intracranial volume (TICV), were used to measure brain parenchymal fraction (BPF). A deep learning algorithm segmented WMH volumes from FLAIR sequences. Volumes were log
resultsSeventy-seven participants (mean age 32 ± 9 years, 43% female) who underwent HSCT were followed for a median 5.3 years (interquartile range [IQR] = 4.6). For 65 participants with successful HSCT, BPF declined nonsignificantly (-0.055%/year; 95% CI -0.12, 0.006; DISCUSSION: Successful HSCT was associated with preserved brain volume, but not WMH growth, another brain health biomarker. Whether these HSCT-related brain structural changes translate to cognitive preservation remains to be determined. TRIAL REGISTRATION INFORMATION: ClinicalTrials.gov NCT02140554 registered 2014-05-14, first enrollment February 2015; ClinicalTrials.gov NCT00061568; registered 2004-07-16, first enrollment July 2004; ClinicalTrials.gov NCT02105766; registered 2014-04-01, first enrollment April 2014; ClinicalTrials.gov NCT03077542; registered 2017-03-10, first enrollment April 2017; ClinicalTrials.gov NCT00977691; registered 2009-12-14, first enrollment December 2009. CLASSIFICATION OF EVIDENCE: This study provides Class III evidence that in adults with sickle cell disease, successful hematopoietic stem cell transplant is associated with slower declines in BPFs over a median period of 5 years, compared with patients with transplant failure.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.