Evidence map›Paper›PMID 23416852›Full record

Trial reportBiology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation2013

Long-term outcome and evaluation of organ function in pediatric patients undergoing haploidentical and matched related hematopoietic cell transplantation for sickle cell disease.

Mari H Dallas, Brandon Triplett, David R Shook, Christine Hartford, Ashok Srinivasan, Joseph Laver, Russell Ware, Wing Leung

Registry-linked trialAbstract readClinical Trial, Phase II
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04523376 (Closely Matched Unrelated Donor Peripheral Blood Stem Cell Transplantation With TCRαβ+ T Cell and B Cell Depletion For Patients With Sickle Cell Disease and Thalassemia Major), which is not on this map. Cited by 72 papers, 4 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
72citing papers in PubMed, 4 pooled it
–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.

NCT04523376 nacompletednot on this mapstarted 2020, after this paper: background citation

Closely Matched Unrelated Donor Peripheral Blood Stem Cell Transplantation With TCRαβ+ T Cell and B Cell Depletion For Patients With Sickle Cell Disease and Thalassemia Major

TypeinterventionalSponsorTimothy OlsonRan2020 to 2025Enrolled8ConditionsSickle Cell Disease, Thalassemia MajorArmsCliniMACS
3 · Its place in the literature

Who cites it

72 citing papers in PubMed, 4 syntheses or guidelines pooled it.

  1. Pooled it
  2. Pooled it
  3. Guideline
  4. Pooled it
  5. Trial
  6. Trial
  7. Trial
  8. Trial
  9. Article
  10. Review
  11. Review
  12. Article
  13. Article
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  15. Review
  16. Article
  17. Haploidentical HSCT in the Treatment of Pediatric Hematological Disorders.International journal of molecular sciences · 2024
    Review
  18. The effectiveness of hematopoietic stem cell transplantation in treating pediatric sickle cell disease: Systematic review and meta-analysis.Saudi pharmaceutical journal : SPJ : the official publication of the Saudi Pharmaceutical Society · 2024
    Review
  19. Review
  20. Article

12 more citing papers are in PubMed but not listed here.

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

8 authors.

Mari H DallasDepartment of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, TN, USA. mari.dallas@stjude.org
Brandon Triplett
David R Shook
Christine Hartford
Ashok Srinivasan
Joseph Laver
Russell Ware
Wing Leung

Funding

NIAID NIH HHS L40 AI071865
6 · The paper itself

Abstract

HLA-matched related donor (MRD) hematopoietic stem cell transplantation (HSCT) is a well-established therapy for patients with sickle cell disease (SCD); however, experience using alternative donors, including haploidentical donors, in HSCT for SCD is limited. We report the long-term outcomes of 22 pediatric patients who underwent related donor HSCT for SCD at St. Jude Children's Research Hospital, either a myeloablative sibling MRD HSCT (n = 14) or reduced-intensity parental haploidentical donor HSCT (n = 8). The median patient age was 11.0 ± 3.9 years in the MRD graft recipients and 9.0 ± 5.0 years in the haploidentical donor graft recipients. The median follow-up was 9.0 ± 2.3 years, with an overall survival (OS) of 93% and a recurrence/graft failure rate of 0%, for the MRD cohort and 7.4 ± 2.4 years, with an OS of 75%, disease-free survival of 38%, and disease recurrence of 38%, for the haploidentical donor cohort. We report the long-term hematologic response and organ function in patients undergoing MRD or haploidentical donor HSCT for severe SCD. Our data demonstrate long-term hematologic improvements after HSCT with sustained engraftment, and confirm that HSCT offers long-term protection from common complications of SCD, including stroke, pulmonary hypertension, acute chest, and nephropathy, regardless of donor source.

Indexed as

AdolescentAnemia, Sickle CellChildChild, PreschoolFemaleHaploidyHematopoietic Stem Cell TransplantationHumansMaleSurvival RateTransplantation, HomologousTreatment Outcome

Identifiers

PMID23416852
PMCPMC4712646

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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.