Evidence map›Paper›PMID 42448317›Full record

ArticleBlood advances2026

Blood rheology after allogeneic hematopoietic stem cell transplantation or gene therapy in sickle cell disease.

Ashwin P Patel, Tami D John, Stephen Gottschalk, Brandon M Triplett, Serena De Vita, Elizabeth O Stenger, Akshay Sharma, Vivien A Sheehan

Abstract read
In one paragraph

Article in Blood advances, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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1 · What the graph read from it

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2 · The registry

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3 · Its place in the literature

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4 · The record

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5 · Who and what money

Authors and funding

8 authors.

Ashwin P PatelAflac Cancer and Blood Disorders Center, Department of Pediatrics, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA.ORCID 0000-0003-0075-3304
Tami D JohnDepartment of Pediatrics, Stanford Medicine Children's Health, Palo Alto, CA.ORCID 0000-0003-0632-3559
Stephen GottschalkDepartment of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, TN.ORCID 0000-0003-3991-7468
Brandon M TriplettDepartment of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, TN.ORCID 0000-0001-8220-9980
Serena De VitaNovartis Biomedical Research, Cambridge, MA.
Elizabeth O StengerAflac Cancer and Blood Disorders Center, Department of Pediatrics, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA.ORCID 0000-0002-1386-6685
Akshay SharmaDepartment of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, TN.ORCID 0000-0003-3281-2081
Vivien A SheehanAflac Cancer and Blood Disorders Center, Department of Pediatrics, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA.ORCID 0000-0003-2837-2255

Funding

Novel therapeutic gene editing to induce fetal hemoglobin for sickle cell diseaseU01HL163983 · NHLBI · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · PI Akshay Sharma, Shengdar Tsai · 2023 to 2026
$4.9M
NHLBI NIH HHS U01 HL163983
6 · The paper itself

Abstract

abstractAllogeneic hematopoietic cell transplantation (HCT) and autologous hematopoietic stem cell-based gene therapies are potentially curative for sickle cell disease (SCD). Although most patients experience resolution of overt clinical symptoms associated with SCD with donor-dominant myeloid chimerism after undergoing HCT or the induction of a substantial amount of alternative hemoglobin (Hb) after gene therapy (GT), whether the underlying subclinical physiology is normalized in these individuals is unknown. One approach to evaluating the underlying physiology is to characterize the blood rheology of patients after they receive these therapies. We assessed the rheology of 123 blood samples from 69 patients, of whom 43 underwent HCT (32 from matched related donors; 11 from haploidentical donors) and 11 received GT. The results were compared to those for patients treated with hydroxyurea with optimum fetal Hb response (HUOptimum; n = 15). Median rheology parameters of recipients of HCT from donors with sickle cell trait (HbAS) were superior to those of GT recipients (elongation index under hypoxia [EImin]: 0.6 vs 0.31; point of sickling [PoS]: 0 vs 26.21) and of the HUOptimum cohort (EImin, 0.6 vs 0.15; PoS, 0 vs 36.2; percentage of dense red blood cells, 0.9 vs 4.9; P< .05 for all comparisons). Results for the GT and HUOptimum groups were comparable (P> .05). Given the substantial number of patients with abnormal rheology and the associations between rheology and SCD-related complications, rheology tests may be included in the follow-up care for HCT and GT recipients.

Indexed as

Anemia, Sickle CellGenetic TherapyHematopoietic Stem Cell TransplantationHemorheologyAdolescentAdultChildFemaleHumansMaleRheologyTransplantation, HomologousYoung Adult

Identifiers

PMID42448317
PMCPMC13583962

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