Evidence map›Paper›PMID 40171203›Full record

ReviewJournal of blood medicine2025

Clinical Outcomes Among Patients With Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia Treated With Allogeneic Hematopoietic Stem Cell Transplantation: A Systematic Literature Review.

Rajeshwari Nair, Nanxin Li, Suzan Imren, Puja Kohli, Krzysztof Lach, Lucía Zhu, Mirella Dudzic

Abstract readReview
In one paragraph

Review in Journal of blood medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
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

2 citing papers in PubMed.

  1. Observational
  2. Review
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

7 authors.

Rajeshwari NairVertex Pharmaceuticals Incorporated, Boston, MA, USA.ORCID 0000-0002-9406-7841
Nanxin LiVertex Pharmaceuticals Incorporated, Boston, MA, USA.
Suzan ImrenVertex Pharmaceuticals Incorporated, Boston, MA, USA.
Puja KohliVertex Pharmaceuticals Incorporated, Boston, MA, USA.
Krzysztof LachMaple Health Group LLC, New York, NY, USA.
Lucía ZhuMaple Health Group LLC, New York, NY, USA.
Mirella DudzicMaple Health Group LLC, New York, NY, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: The purpose of this study is to synthesize evidence on disease-specific outcomes in patients with sickle cell disease (SCD) or transfusion-dependent beta-thalassemia (TDT) following allogeneic hematopoietic stem cell transplant (allo-HSCT). Methods: A systematic literature review (SLR) was conducted in MEDLINE and Embase to identify publications up to May 2023, including patients with SCD or TDT treated with allo-HSCT. Occurrence of vaso-occlusive crises (VOCs) including acute pain, acute chest syndrome, priapism, and splenic sequestration in SCD, and red blood cell transfusion (RBCT) requirements in TDT were the main outcomes of interest. Transplant-related outcomes such as graft-versus-host disease (GVHD) and graft failure/rejection were summarized in the studies that reported main outcomes. Proportion of patients experiencing VOCs or RBCTs, GVHD, and graft failure/rejection after allo-HSCT were aggregated and descriptively reported with range across studies. Results: Thirty-one SCD studies met inclusion criteria. Twenty-nine studies assessed for VOC and pain crisis events after allo-HSCT; 11 studies reported ≥1 VOCs after allo-HSCT in 6.9% of the 2,760 patients. Graft failure was reported in 14.4% (0.9%-18.8%, 14 studies) of patients, graft rejection in 5.5% (1.6%-100.0%, 12 studies) of patients, acute GVHD in 22.4% (1.6%-50.0%, 19 studies) of patients, and chronic GVHD in 20.4% (3.3%-57.1%, 14 studies) of patients. Seventy-eight TDT studies met inclusion criteria. Fifty-six studies reported that 8.8% of the 3,107 patients required RBCTs after allo-HSCT. Graft failure was reported in 5.4% (1.1%-80.0%, 21 studies) of patients, graft rejection in 7.5% (0.5%-42.9%, 50 studies) of patients, acute GVHD in 28.4% (5.2%-100.0%, 57 studies) and chronic GVHD in 15.2% (1.3%-50.0%, 51 studies) of TDT patients. Conclusion: Based on this SLR, after allo-HSCT, a portion of patients with SCD continue to experience VOCs and a portion of patients with TDT continue to require RBCTs, in addition to experiencing GVHD and graft failure or rejection.

Indexed as

allogeneic HSCTbeta-thalassemiasickle cell diseasetransfusion dependentvaso-occlusive crisis

Identifiers

PMID40171203
PMCPMC11960486

What OpenQuestion holds

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Registered trials

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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.