Evidence map›Paper›PMID 41904735›Full record

Trial reportJournal of clinical immunology2026

Continuous Infusion of the CXCR4 Antagonist Plerixafor for WHIM Syndrome.

David H McDermott, Shamik Majumdar, Daniel Velez, Elena Cho, Zhanzhou Li, Ji-Liang Gao, Megan C Grieco, Monica G Lawrence, Susana L Silva, Leslie A Castelo-Soccio and 2 more

Abstract readClinical Trial, Phase IIClinical Trial, Phase I
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Journal of clinical immunology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. Non-Malignant Granulocyte and Monocyte Disorders: An Update.British journal of biomedical science · 2025
    Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

12 authors.

David H McDermottLaboratory of Molecular Immunology, National Institute of Allergy and Infectious Diseases, National Institutes of Health (NIH), Bethesda, MD, USA.
Shamik MajumdarLaboratory of Molecular Immunology, National Institute of Allergy and Infectious Diseases, National Institutes of Health (NIH), Bethesda, MD, USA.
Daniel VelezLaboratory of Molecular Immunology, National Institute of Allergy and Infectious Diseases, National Institutes of Health (NIH), Bethesda, MD, USA.
Elena ChoLaboratory of Molecular Immunology, National Institute of Allergy and Infectious Diseases, National Institutes of Health (NIH), Bethesda, MD, USA.
Zhanzhou LiLaboratory of Molecular Immunology, National Institute of Allergy and Infectious Diseases, National Institutes of Health (NIH), Bethesda, MD, USA.
Ji-Liang GaoLaboratory of Molecular Immunology, National Institute of Allergy and Infectious Diseases, National Institutes of Health (NIH), Bethesda, MD, USA.
Megan C GriecoBiostatics Research Branch, Division of Clinical Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
Monica G LawrenceDivision of Asthma, Allergy and Immunology, University of Virginia School of Medicine, Charlottesville, VA, USA.
Susana L SilvaFaculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal.
Leslie A Castelo-SoccioOffice of the Director, National Institute of Arthritis and Musculoskeletal and Skin Diseases, National Institutes of Health, Bethesda, MD, USA.
Dean FollmannBiostatics Research Branch, Division of Clinical Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
Philip M MurphyLaboratory of Molecular Immunology, National Institute of Allergy and Infectious Diseases, National Institutes of Health (NIH), Bethesda, MD, USA. pmm@nih.gov.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundWHIM (Warts, Hypogammaglobulinemia, Infections and Myelokathexis) syndrome is an ultrarare inborn error of immunity caused by heterozygous, hyperfunctional CXCR4 mutations that impede leukocyte egress from bone marrow, resulting in panleukopenia. The CXCR4 antagonist plerixafor (AMD3100, Mozobil) durably reverses panleukopenia and in most WHIM patients also induces wart regression; however, its short half-life requires twice daily injection.

objectiveThe aim of this study was to assess the safety and efficacy of continuously infused plerixafor in patients with WHIM syndrome.

methodsWe conducted a Phase 1/2 pilot study of WHIM patients given plerixafor 0.02–0.08 mg/kg/d by continuous subcutaneous infusion for 1–2 years using an OmniPod insulin pump, and assessed compliance as well as effects on blood leukocyte counts, infections, chronic skin conditions and adverse events.

resultsSix patients were treated for a total of 6.3 patient-years; one patient dropped out early for personal reasons related to taking a new job. The drug infusion rate was adjusted to achieve a normal absolute lymphocyte count and an absolute neutrophil count > 500 cells/µl in all patients. An average of 2.1 infections/patient-year occurred (range 0–4). Treatment of two infections involved brief hospitalization. On plerixafor, partial wart regression occurred in 3 of 4 patients, a single molluscum contagiosum infection regressed and a chronic post-Mohs surgical wound epithelialized. There were 3 serious adverse events, but none was attributable to the treatment. All patients preferred pump administration over syringe injection.

conclusionIn WHIM patients a continuous infusion pump may be a convenient, safe and potentially effective means of delivering plerixafor chronically to correct panleukopenia and to improve chronic skin conditions.

Indexed as

Heterocyclic CompoundsImmunologic Deficiency SyndromesPrimary Immunodeficiency DiseasesReceptors, CXCR4WartsAdolescentAdultBenzylaminesCyclamsFemaleHumansMaleMiddle AgedPilot ProjectsTreatment OutcomeYoung AdultBenzylaminesCXCR4 protein, humanCyclamsHeterocyclic CompoundsplerixaforReceptors, CXCR4Chemokine receptorClinical trialImmunodeficiencyLymphopeniaNeutropenia

Identifiers

PMID41904735
PMCPMC13152970

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

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.