Evidence map›Paper›PMID 38181784›Full record

Trial reportBlood advances2024

In vivo measurement of RBC survival in patients with sickle cell disease before or after hematopoietic stem cell transplantation.

Alexis K Leonard, Dana Furstenau, Zaina Inam, Christina Luckett, Rebecca Chu, Selami Demirci, Khaled Essawi, Bjorg Gudmundsdottir, Malikiya Hinds, Julia DiNicola and 11 more

Registry-linked trialOpen access · goldAbstract readClinical Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Blood advances, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04476277 (Red Cell Half Life Determination in Patients With and Without Sickle Cell Disease), which is not on this map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
2.6field-weighted citation impact, top 11% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

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.

NCT04476277 early_phase1completednot on this map

Red Cell Half Life Determination in Patients With and Without Sickle Cell Disease

TypeinterventionalSponsorNational Heart, Lung, and Blood Institute (NHLBI)Ran2021 to 2023Enrolled22ConditionsSickle Cell Disease, Sickle Cell AnemiaArmsBiotin label
3 · Its place in the literature

Who cites it

4 citing papers in PubMed, 6 citations in OpenAlex.

  1. Article
  2. Article
  3. Review
  4. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

21 authors at 4 institutions in 2 countries.

Alexis K LeonardCellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD.
Dana FurstenauCellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD.
Zaina InamCellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD.ORCID 0000-0003-0022-4138
Christina LuckettCellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD.
Rebecca ChuCellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD.
Selami DemirciCellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD.ORCID 0000-0002-6174-1609
Khaled EssawiDepartment of Medical Laboratory Technology, College of Applied Medical Sciences, Jazan University, Gizan, Saudi Arabia.ORCID 0009-0003-9556-5249
Bjorg GudmundsdottirCellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD.
Malikiya HindsCellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD.
Julia DiNicolaCellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD.
Quan LiLaboratory of Chemical Physics, National Institute of Diabetes and Digestive and Kidney Diseases, Bethesda, MD.
William A EatonLaboratory of Chemical Physics, National Institute of Diabetes and Digestive and Kidney Diseases, Bethesda, MD.ORCID 0000-0002-9244-5407
Troy CellmerLaboratory of Chemical Physics, National Institute of Diabetes and Digestive and Kidney Diseases, Bethesda, MD.
Xunde WangSickle Cell Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD.
Swee Lay TheinSickle Cell Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD.ORCID 0000-0002-9835-6501
Elizabeth R Macaribluebird bio, Inc, Somerville, MA.
Sara VanNestbluebird bio, Inc, Somerville, MA.
Matthew M HsiehCellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD.
Melissa Bonnerbluebird bio, Inc, Somerville, MA.
Francis J Piercieybluebird bio, Inc, Somerville, MA.
John F TisdaleCellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD.
National Institutes of Health · USBluebird Bio (United States) · USNational Institute of Diabetes and Digestive and Kidney Diseases · USJazan University · SA

Funding

Sickle cell disease molecular pathogenesis and drug therapyZIADK029053 · NIDDK · NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES · PI EATON, WILLIAM · 2009 to 2025
$24.8M
LAB RESEARCH TRAINING IN PEDIATRIC ONCOLOGY-HEMATOLOGYT32CA060441 · NCI · JOHNS HOPKINS UNIVERSITY · PI ALAN D FRIEDMAN · 1993 to 2026
$14.9M
NCI NIH HHS T32 CA060441
6 · The paper itself

Abstract

abstractStable, mixed-donor-recipient chimerism after allogeneic hematopoietic stem cell transplantation (HSCT) for patients with sickle cell disease (SCD) is sufficient for phenotypic disease reversal, and results from differences in donor/recipient-red blood cell (RBC) survival. Understanding variability and predictors of RBC survival among patients with SCD before and after HSCT is critical for gene therapy research which seeks to generate sufficient corrected hemoglobin to reduce polymerization thereby overcoming the red cell pathology of SCD. This study used biotin labeling of RBCs to determine the lifespan of RBCs in patients with SCD compared with patients who have successfully undergone curative HSCT, participants with sickle cell trait (HbAS), and healthy (HbAA) donors. Twenty participants were included in the analysis (SCD pre-HSCT: N = 6, SCD post-HSCT: N = 5, HbAS: N = 6, and HbAA: N = 3). The average RBC lifespan was significantly shorter for participants with SCD pre-HSCT (64.1 days; range, 35-91) compared with those with SCD post-HSCT (113.4 days; range, 105-119), HbAS (126.0 days; range, 119-147), and HbAA (123.7 days; range, 91-147) (P<.001). RBC lifespan correlated with various hematologic parameters and strongly correlated with the average final fraction of sickled RBCs after deoxygenation (P<.001). No adverse events were attributable to the use of biotin and related procedures. Biotin labeling of RBCs is a safe and feasible methodology to evaluate RBC survival in patients with SCD before and after HSCT. Understanding differences in RBC survival may ultimately guide gene therapy protocols to determine hemoglobin composition required to reverse the SCD phenotype as it relates directly to RBC survival. This trial was registered at www.clinicaltrials.gov as #NCT04476277.

Indexed as

Anemia, Sickle CellHematopoietic Stem Cell TransplantationBiotinErythrocytesHemoglobinsHumansBiotinHemoglobins

Identifiers

PMID38181784
PMCPMC11006808
OpenAlexW4390605785

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

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.