Evidence map›Paper›PMID 41741059›Full record

ReviewClinics in laboratory medicine2026

Emerging Considerations in Transfusion Medicine: Hematopoietic Stem and Progenitor Cell Collection for Gene Therapy for Sickle Cell Disease and Transfusion-Dependent Thalassemia.

Nora M Gibson, Janet L Kwiatkowski, Stella T Chou

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Clinics in laboratory medicine, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

3 authors.

Nora M GibsonDivision of Oncology, Department of Pediatrics, Children's Hospital of Philadelphia, Colket Translational Research Building, Room 4020, 3501 Civic Center Boulevard, Philadelphia, PA 19104, USA.
Janet L KwiatkowskiDivision of Hematology, Department of Pediatrics, Children's Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, 3500 Civic Center Boulevard, Philadelphia, PA 19104, USA.
Stella T ChouDivision of Hematology, Department of Pediatrics, Children's Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, 3500 Civic Center Boulevard, Philadelphia, PA 19104, USA; Division of Transfusion Medicine, Children's Hospital of Philadelphia, 3400 Civic Center Boulevard, Philadelphia, PA 19104, USA. Electronic address: chous@chop.edu.

Funding

Improving transfusion therapy for patients with sickle cell disease with pluripotent stem cell-derived red cellsU01HL134696 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI CHOU, STELLA T · 2016 to 2022
$8.3M
RED CELL HEMOGLOBIN RESEARCH TRAINING PROGRAMT32HL007150 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI STELLA T CHOU, Mortimer Poncz · 1985 to 2026
$7.4M
A precision therapy approach to red cell transfusion for sickle cell diseaseR01HL169401 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI STELLA T CHOU · 2024 to 2026
$2.4M
Manufacturing Process Intensification for Pluripotent Stem Cell-Derived Rare Red Cell ProductionR41HL178144 · NHLBI · SAFI BIOTHERAPEUTICS INC · PI CHOU, STELLA T, THOMAS, ROBERT JAMES · 2025 to 2025
$568k
NHLBI NIH HHS R01 HL169401NHLBI NIH HHS R41 HL178144NHLBI NIH HHS T32 HL007150NHLBI NIH HHS U01 HL134696
6 · The paper itself

Abstract

Gene-modified hematopoietic stem cell transplant (GM-HSCT) is an innovative curative therapy for transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD), which has demonstrated excellent efficacy in clinical trials. Two commercial products are currently available, with several additional products in clinical trials. GM-HSCT requires the mobilization and subsequent collection of hematopoietic stem and progenitor cells (HSPCs) by apheresis. Current protocols consistently demonstrate effectiveness in collecting HSPCs in patients with TDT. However, disease-specific characteristics of SCD create challenges in collecting HSPCs. Further research remains in optimizing collection procedures in this population.

Indexed as

Gene therapyHematopoietic stem and progenitor cell collectionSickle cell diseaseTransfusion-dependent thalassemia

Identifiers

PMID41741059
PMCPMC13454772

What OpenQuestion holds

Textmetadata
LicenceTDM
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.