Evidence map›Paper›PMID 36240296›Full record

ArticleBlood advances2023

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

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

Open access · goldAbstract read
In one paragraph

Article in Blood advances, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
18citing papers in PubMed, 2 pooled it
2.6field-weighted citation impact, top 9% 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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

18 citing papers in PubMed, 2 syntheses or guidelines pooled it, 19 citations in OpenAlex.

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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors at 2 institutions in 1 country.

Alexis LeonardCellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD.ORCID 0000-0003-4317-3879
Dana FurstenauCellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD.
Allistair AbrahamCenter for Cancer and Immunology Research, CETI, Children's National Hospital, Washington, DC.ORCID 0000-0003-2019-0887
Deepika S DarbariDivision of Hematology, Children's National Hospital, Washington, DC.
Robert S NickelDivision of Hematology, Children's National Hospital, Washington, DC.ORCID 0000-0003-4277-7523
Emily LimerickCellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD.
Courtney FitzhughCellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD.ORCID 0000-0002-5306-7167
Matt HsiehCellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD.
John F TisdaleCellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD.ORCID 0000-0002-8535-9439
National Institutes of Health · USChildren's National · US

Funding

Nonmyeloablative haploidentical peripheral blood stem cell transplantation in congenital anemiasZIAHL006211 · NHLBI · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · PI FITZHUGH, COURTNEY · 2016 to 2025
$19.4M
Nonmyeloablative allogeneic PBSC in globin disordersZIAHL006007 · NHLBI · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · PI TISDALE, JOHN · 2009 to 2025
$18.4M
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

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.

Indexed as

Anemia, Sickle CellHematopoietic Stem Cell TransplantationGraft RejectionHumansInfantTransplantation, AutologousTransplantation Conditioning

Identifiers

PMID36240296
PMCPMC9860452
OpenAlexW4306194570

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

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.