Evidence map›Paper›PMID 25636378›Full record

ArticleBiology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation2015

Matched related and unrelated donor hematopoietic stem cell transplantation for DOCK8 deficiency.

Jennifer Cuellar-Rodriguez, Alexandra F Freeman, Jennifer Grossman, Helen Su, Mark Parta, Heardley Murdock, Nirali Shah, Catherine Bollard, Heidi H Kong, Niki Moutsopoulos and 4 more

Abstract readClinical Trial, Phase I
In one paragraph

Article in Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 33 papers.

0numbers the graph read from it
0cells of the map it votes in
33citing 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

33 citing papers in PubMed.

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  11. Clinical, immunological features and follow up of 20 patients with dedicator of cytokinesis 8 (DOCK8) deficiency.Pediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology · 2020
    Article
  12. Article
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  14. Hematopoietic Stem Cell Transplantation as Treatment for Patients with DOCK8 Deficiency.The journal of allergy and clinical immunology. In practice · 2019
    Article
  15. Review
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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

14 authors.

Jennifer Cuellar-RodriguezLaboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland.
Alexandra F FreemanLaboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland.
Jennifer GrossmanDivision of Hematology and Hematologic Malignancies, Alberta Health Services, Calgary, Alberta, Canada.
Helen SuLaboratory of Host Defense, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland.
Mark PartaClinical Research Directorate/Clinical Monitoring Research Program, Leidos Biomedical Research, Inc. (formerly SAIC-Frederick, Inc.), Frederick National Laboratory for Cancer Research, Frederick, Maryland.
Heardley MurdockLaboratory of Host Defense, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland.
Nirali ShahPediatric Oncology Branch, National Cancer Institute, National Institutes of Health, Bethesda, Maryland.
Catherine BollardCenter for Cancer and Immunology Research at the Children's Research Institute, Children's National Medical Center, Washington, DC.
Heidi H KongDermatology Branch, National Cancer Institute, National Institutes of Health, Bethesda, Maryland.
Niki MoutsopoulosNational Institute of Dental and Craniofacial Research, National Institutes of Health, Bethesda, Maryland.
Kelly StoneLaboratory of Allergic Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland.
Juan Gea-BanaclocheExperimental Transplantation and Immunology Branch, National Cancer Institute, National Institutes of Health, Bethesda, Maryland.
Steven M HollandLaboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland.
Dennis D HicksteinExperimental Transplantation and Immunology Branch, National Cancer Institute, National Institutes of Health, Bethesda, Maryland. Electronic address: hicksted@mail.nih.gov.

Funding

Genes And Gene Products As ImmunoadjuvantsZIAAI000647 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI HOLLAND, STEVEN · 2009 to 2025
$22.6M
Transgenic Animal Models Of Human Immune DefectsZIAAI000646 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI HOLLAND, STEVEN · 2009 to 2025
$22.2M
Oral Microbial and Immunological Characterization of Patients with Immune DysfunZIADE000732 · NIDCR · NATIONAL INSTITUTE OF DENTAL & CRANIOFACIAL RESEARCH · PI MOUTSOPOULOS, NIKI · 2010 to 2024
$13.2M
Effects of systemic and topical perturbations on human microbesZIABC011558 · NCI · DIVISION OF BASIC SCIENCES - NCI · PI KONG, HEIDI · 2014 to 2021
$6.9M
Pathogenesis and Treatment of Atopic DermatitisZIAAI001098 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI MILNER, JOSHUA · 2009 to 2019
$6.5M
Studies of Skin Microflora in Healthy Individuals and Atopic Dermatitis PatientsZIABC010938 · NCI · DIVISION OF BASIC SCIENCES - NCI · PI KONG, HEIDI · 2009 to 2021
$5.4M
Understanding DOCK8 Function in Health and Human DiseaseZIAAI001193 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI SU, HELEN CHUN-HUI · 2014 to 2025
$4.5M
Allotransplant for DOCK8 deficiencyZIABC011374 · NCI · DIVISION OF BASIC SCIENCES - NCI · PI HICKSTEIN, DENNIS · 2011 to 2020
$3.0M
CCR NIH HHS HHSN261200800001CIntramural NIH HHS ZIA BC011374NCI NIH HHS HHSN261200800001E
6 · The paper itself

Abstract

We performed allogeneic hematopoietic stem cell transplantation in 6 patients with mutations in the dedicator-of-cytokinesis-8 (DOCK8) gene using a myeloablative conditioning regimen consisting of busulfan 3.2 mg/kg/day i.v. for 4 days and fludarabine 40 mg/m(2)/day for 4 days. Three patients received allografts from matched related donors and 3 patients from matched unrelated donors. Two patients received peripheral blood stem cells and 4 patients bone marrow hematopoietic stem cells. Tacrolimus and short-course methotrexate on days 1, 3, 6, and 11 were used for graft-versus-host-disease (GVHD) prophylaxis. All 6 patients are alive at a median follow-up of 22.5 months (range, 14 to 35). All patients achieved rapid and high levels of donor engraftment and complete reversal of the clinical and immunologic phenotype. Adverse events consisted of acute skin GVHD in 2 patients and post-transplant pulmonary infiltrates in a patient with extensive bronchiectasis pretransplant. Thus, a uniform myeloablative conditioning regimen followed by allogeneic hematopoietic stem cell transplantation in DOCK8 deficiency results in reconstitution of immunologic function and reversal of the clinical phenotype with a low incidence of regimen-related toxicity.

Indexed as

Hematopoietic Stem Cell TransplantationAdolescentAdultBusulfanChildFemaleGraft SurvivalGraft vs Host DiseaseGuanine Nucleotide Exchange FactorsHistocompatibility TestingHumansImmunophenotypingImmunosuppressive AgentsMaleMethotrexateMutationBusulfanDOCK8 protein, humanfludarabineGuanine Nucleotide Exchange FactorsImmunosuppressive AgentsMethotrexateMyeloablative AgonistsTacrolimusVidarabineAllogeneicDOCK8 deficiencyTransplantation

Identifiers

PMID25636378
PMCPMC4426076

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Registered trials

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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.