Evidence map›Paper›PMID 22955919›Full record

Trial reportBlood2012

HLA-haploidentical bone marrow transplantation with posttransplant cyclophosphamide expands the donor pool for patients with sickle cell disease.

Javier Bolaños-Meade, Ephraim J Fuchs, Leo Luznik, Sophie M Lanzkron, Christopher J Gamper, Richard J Jones, Robert A Brodsky

2 registry-linked trialsOpen access · bronzeAbstract readClinical Trial, Phase IClinical Trial, Phase II
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Blood, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 221 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
221citing papers in PubMed, 3 pooled it
16.2field-weighted citation impact, top 1% 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.

NCT03240731 phase2completednot on this mapstarted 2017, after this paper: background citation

Bone Marrow Transplantation HLA Haploidentical After a Reduced Intensity Conditioning and Prevention of GvHD Based on Post-transplant Cyclophosphamide Administration in Patients With Severe Sickle Cell Disease

TypeinterventionalSponsorCentre Hospitalier Intercommunal CreteilRan2017 to 2024Enrolled25ConditionsSickle Cell DiseaseArmsbone marrow transplant
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

221 citing papers in PubMed, 3 syntheses or guidelines pooled it, 437 citations in OpenAlex.

  1. Guideline
  2. Pooled it
  3. Psychological therapies for sickle cell disease and pain.The Cochrane database of systematic reviews · 2015
    Pooled it
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  18. Unrelated Donor Transplantation in Children with Thalassemia using Reduced-Intensity Conditioning: The URTH Trial.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2018
    Trial
  19. Trial
  20. Haploidentical stem cell transplantation with CD3Bone marrow transplantation · 2017
    Trial

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

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

7 authors at 3 institutions in 1 country.

Javier Bolaños-MeadeHematologic Malignancies and Bone Marrow Transplantation Program, Department of Oncology, Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins and Johns Hopkins University School of Medicine, Baltimore, MD 21231-1000, USA. fbolano2@jhmi.edu
Ephraim J Fuchs
Leo Luznik
Sophie M Lanzkron
Christopher J Gamper
Richard J Jones
Robert A Brodsky
Johns Hopkins University · USSidney Kimmel Comprehensive Cancer Center · USJohns Hopkins Medicine · US

Funding

TUMOR VACCINESP01CA015396 · NCI · JOHNS HOPKINS UNIVERSITY · PI JONES, RICHARD J · 1985 to 2016
$38.1M
Sickle Cell Disease: First steps toward understanding barriers to care for adultsK23HL083089 · NHLBI · JOHNS HOPKINS UNIVERSITY · PI LANZKRON, SOPHIE M · 2008 to 2012
$701k
NCI NIH HHS P01 CA015396NCI NIH HHS P01CA15396NHLBI NIH HHS K23 HL083089NHLBI NIH HHS K23HL083089
6 · The paper itself

Abstract

Allogeneic marrow transplantation can cure sickle cell disease; however, HLA-matched donors are difficult to find, and the toxicities of myeloablative conditioning are prohibitive for most adults with this disease. We developed a nonmyeloablative bone marrow transplantation platform using related, including HLA-haploidentical, donors for patients with sickle cell disease. The regimen consisted of antithymocyte globulin, fludarabine, cyclophosphamide, and total body irradiation, and graft-versus-host disease prophylaxis with posttransplantation high-dose cyclophosphamide, mycophenolate mofetil, and tacrolimus or sirolimus. After screening 19 patients, we transplanted 17, 14 from HLA-haploidentical and 3 from HLA-matched related donors. Eleven patients engrafted durably. With a median follow-up of 711 days (minimal follow up 224 days), 10 patients are asymptomatic, and 6 patients are off immunosupression. Only 1 patient developed skin-only acute graft-versus-host disease that resolved without any therapy; no mortality was seen. Nonmyeloablative conditioning with posttransplantation high-dose cyclophosphamide expands the donor pool, making marrow transplantation feasible for most patients with sickle cell disease, and is associated with a low risk of complications, even with haploidentical related donors. Graft failure, 43% in haploidentical pairs, remains a major obstacle but may be acceptable in a fraction of patients if the majority can be cured without serious toxicities.

Indexed as

AdolescentAdultAnemia, Sickle CellBone Marrow TransplantationCyclophosphamideDrug Administration ScheduleFemaleHistocompatibilityHistocompatibility TestingHumansImmunosuppressive AgentsMaleMiddle AgedTissue DonorsTransplantation ConditioningTreatment OutcomeCyclophosphamideImmunosuppressive Agents

Identifiers

PMID22955919
PMCPMC3507140
OpenAlexW2001175629

What OpenQuestion holds

Textmetadata
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.