Evidence map›Paper›PMID 33534948›Full record

Trial reportBritish journal of haematology2021

Non-myeloablative human leukocyte antigen-matched related donor transplantation in sickle cell disease: outcomes from three independent centres.

Mohsen Alzahrani, Moussab Damlaj, Neal Jeffries, Bader Alahmari, Avani Singh, Damiano Rondelli, John F Tisdale, Santosh L Saraf, Matthew M Hsieh

Open access · greenAbstract readClinical TrialMulticenter Study
In one paragraph

Trial report in British journal of haematology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 42 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
42citing papers in PubMed, 1 pooled it
6.6field-weighted citation impact, top 2% 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

42 citing papers in PubMed, 1 synthesis or guideline pooled it, 62 citations in OpenAlex.

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  19. Hematopoietic Stem Cell Transplantation in Sickle Cell Disease.Advances in experimental medicine and biology · 2025
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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

9 authors at 3 institutions in 2 countries.

Mohsen AlzahraniDivision of Hematology & Hematopoietic Stem Cell Transplantation, Department of Oncology, King Abdulaziz Medical City, Riyadh, Saudi Arabia.
Moussab DamlajDivision of Hematology & Hematopoietic Stem Cell Transplantation, Department of Oncology, King Abdulaziz Medical City, Riyadh, Saudi Arabia.
Neal JeffriesOffice of Biostatistics Research, National Heart, Lung, and Blood Institute, Bethesda, MD, USA.
Bader AlahmariDivision of Hematology & Hematopoietic Stem Cell Transplantation, Department of Oncology, King Abdulaziz Medical City, Riyadh, Saudi Arabia.
Avani SinghDivision of Hematology/Oncology, University of Illinois Hospital and Health Science Systems, Chicago, IL, USA.
Damiano RondelliDivision of Hematology/Oncology, University of Illinois Hospital and Health Science Systems, Chicago, IL, USA.
John F TisdaleCellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute, Bethesda, MD, USA.
Santosh L SarafDivision of Hematology/Oncology, University of Illinois Hospital and Health Science Systems, Chicago, IL, USA.ORCID 0000-0002-8584-4194
Matthew M HsiehCellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute, Bethesda, MD, USA.ORCID 0000-0002-3706-6615
King Abdulaziz Medical City · SANational Heart Lung and Blood Institute · USUniversity of Illinois Chicago · US

Funding

Nonmyeloablative allogeneic PBSC in globin disordersZIAHL006007 · NHLBI · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · PI TISDALE, JOHN · 2009 to 2025
$18.4M
Intramural NIH HHS Z99 HL999999NHLBI NIH HHS HL006007-11
6 · The paper itself

Abstract

Non-myeloablative haematopoietic progenitor cell transplantation (HPCT) from matched related donors (MRD) has been increasingly utilized in sickle cell disease (SCD). A total of 122 patients received 300 cGy of total body irradiation (TBI), alemtuzumab, unmanipulated filgrastim-mobilized peripheral blood HPC and sirolimus. The median follow-up was four years; median age at HPCT was 29 years. Median neutrophil and platelet engraftment occurred on day 22 and 19 respectively; 41 patients required no platelet transfusions. Overall and sickle-free survival at one and five years were 93% and 85% respectively. Age, sex, pre-HPCT sickle complications, ferritin and infused HPC numbers were similar between graft failure and engrafted patients. Mean donor myeloid chimaerism at one and five years post HPCT were 84% and 88%, and CD3 was 48% and 53% respectively. Two patients developed grade 1 and 2 skin graft-versus-host disease (GVHD) with no chronic GVHD. Median days of recipients taking immunosuppression were 489; 83% of engrafted patients have discontinued immunosuppression. Haemoglobin, haemolytic parameters and hepatic iron levels improved post HPCT. Pulmonary function testing, hepatic histology and neurovascular imaging remained stable, suggesting cessation of further sickle-related injury. Fourteen patients had children. In this largest group of adult SCD patients, this regimen was highly efficacious, well-tolerated despite compromised organ functions pre HPCT, and without clinically significant GVHD.

Indexed as

Hematopoietic Stem Cell TransplantationHLA AntigensAdolescentAdultAlemtuzumabAnemia, Sickle CellAntineoplastic Agents, ImmunologicalChildFemaleGraft RejectionGraft SurvivalGraft vs Host DiseaseHumansImmunosuppressive AgentsMaleMiddle AgedAlemtuzumabAntineoplastic Agents, ImmunologicalHLA AntigensImmunosuppressive AgentsSirolimusalemtuzumaballogeneic transplantationmatched related donorsickle cell diseasesirolimus

Identifiers

PMID33534948
PMCPMC8582053
OpenAlexW3126636560

What OpenQuestion holds

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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.