Evidence map›Paper›PMID 25143186›Full record

Trial reportTransfusion2015

Mobilization characteristics and strategies to improve hematopoietic progenitor cell mobilization and collection in patients with chronic granulomatous disease and severe combined immunodeficiency.

Sandhya R Panch, Yu Ying Yau, Elizabeth M Kang, Suk See De Ravin, Harry L Malech, Susan F Leitman

Registry-linked trialOpen access · greenAbstract readClinical Trial
In one paragraph

Trial report in Transfusion, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03055247 (A Multicentric, Exploratory, Non-randomised, Non-controlled, Prospective, Open-label Phase II Study Evaluating Safety and Efficacy of IBU, G-CSF and Plerixafor as Stem Cell Mobilization Regimen in Patients Affected by X-CGD), which is not on this map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
0.9field-weighted citation impact, top 26% 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.

NCT03055247 phase2recruitingnot on this map

A Multicentric, Exploratory, Non-randomised, Non-controlled, Prospective, Open-label Phase II Study Evaluating Safety and Efficacy of IBU, G-CSF and Plerixafor as Stem Cell Mobilization Regimen in Patients Affected by X-CGD

TypeinterventionalSponsorIRCCS San RaffaeleRan2015 to 2026Enrolled3ConditionsChronic Granulomatous Disease X-linked (X-CGD)ArmsIbuprofen, Myelostim, Mozobil
3 · Its place in the literature

Who cites it

3 citing papers in PubMed, 11 citations in OpenAlex.

  1. Trial
  2. Review
  3. Primary immune deficiencies with defects in neutrophil function.Hematology. American Society of Hematology. Education Program · 2016
    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

6 authors at 2 institutions in 1 country.

Sandhya R PanchHematology/Transfusion Medicine, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland.
Yu Ying Yau
Elizabeth M Kang
Suk See De Ravin
Harry L Malech
Susan F Leitman
National Institutes of Health · USNational Institutes of Health Clinical Center · US

Funding

Development and conduct of allogeneic stem cell transplant and autologous stem cell gene therapy for inherited immune deficienciesZIAAI000989 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI KANG, ELIZABETH · 2009 to 2025
$12.4M
Gene Therapy Basic Research to Treat Inherited Primary Immune DeficienciesZIAAI000644 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI MALECH, HARRY L · 2009 to 2025
$12.1M
Development and Clinical Application of Gene and Cell therapies for Patients with Immune DeficienciesZIAAI000645 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI DE RAVIN, SUK SEE · 2009 to 2025
$10.1M
Basic Research on Hematopoietic Human Stem CellsZIAAI000988 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI MALECH, HARRY L · 2009 to 2019
$6.5M
Gene Therapy Clinical Trials for X-linked Severe Combined ImmunodeficiencyZIAAI000992 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI DE RAVIN, SUK · 2009 to 2019
$4.1M
Gene Therapy Clinical Trials for Chronic Granulomatous DiseaseZIAAI000991 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI KANG, ELIZABETH · 2009 to 2019
$3.4M
Intramural NIH HHS Z99 CL999999
6 · The paper itself

Abstract

backgroundGranulocyte-colony-stimulating factor (G-CSF)-mobilized autologous hematopoietic progenitor cells (HPCs) may be collected by apheresis of patients with chronic granulomatous disease (CGD) and severe combined immunodeficiency (SCID) for use in gene therapy trials. CD34+ cell mobilization has not been well characterized in such patients. STUDY DESIGN AND

methodsWe retrospectively evaluated CD34+ cell mobilization and collection in 73 consecutive CGD and SCID patients and in 99 age-, weight-, and G-CSF dose-matched healthy allogeneic controls.

resultsIn subjects aged not more than 20 years, Day 5 preapheresis circulating CD34+ counts were significantly lower in CGD and SCID patients than in controls; mean peak CD34+ cell counts were 58 × 10(6) , 64 × 10(6) , and 87 × 10(6) /L, respectively (p = 0.01). The SCIDs had lower CD34+ collection efficiency than CGDs and controls; mean efficiencies were 40, 63, and 57%, respectively (p = 0.003). In subjects aged more than 20 years, the CGDs had significantly lower CD34+ cell mobilization than controls; mean peak CD34+ cell counts were 41 × 10(6) and 113 × 10(6) /L, respectively (p < 0.0001). In a multivariate analysis, lower erythrocyte sedimentation rate (ESR) at mobilization was significantly correlated with better CD34+ cell mobilization (p = 0.007). In SCIDs, CD34 collection efficiency was positively correlated with higher red blood cell (RBC) indices (mean RBC volume, R(2)  = 0.77; mean corpuscular hemoglobin [Hb], R(2)  = 0.94; mean corpuscular Hb concentration, R(2)  = 0.7; p < 0.007) but not Hb.

conclusionsCGD and SCID populations are characterized by significantly less robust CD34+ HPC mobilization than healthy controls. The presence of active inflammation or infection as suggested by an elevated ESR may negatively impact mobilization. Among SCIDs, markedly reduced CD34 collection efficiencies were related to iron deficiency, wherein decreased RBC size and density may impair apheresis cell separation mechanics.

Indexed as

Hematopoietic Stem Cell TransplantationAdolescentAdultAutograftsBlood Component RemovalChildFemaleGranulocyte Colony-Stimulating FactorGranulomatous Disease, ChronicHematopoietic Stem Cell MobilizationHumansMaleRetrospective StudiesSevere Combined ImmunodeficiencyGranulocyte Colony-Stimulating Factor

Identifiers

PMID25143186
PMCPMC4331265
OpenAlexW1936895813

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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.