Evidence map›Paper›PMID 39348667›Full record

ArticleBlood advances2024

Efficacy and safety of TPO receptor agonists in treatment of ITP associated with predominantly antibody deficiencies.

Margaux Soulard, Lionel Galicier, Nizar Mahlaoui, Claire Fieschi, Samuel Deshayes, Delphine Gobert, Clément Gourguechon, Hélène Henique, Sebastien Humbert, Carole Lacout and 7 more

Abstract read
In one paragraph

Article in Blood advances, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Article
  2. 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

17 authors.

Margaux SoulardDepartment of Internal Medicine, University Hospital of Rennes, Rennes, France.
Lionel GalicierDepartment of Clinical Immunology, and Immunodeficiencies Center, Saint-Louis Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.
Nizar MahlaouiDepartment of Pediatric Immunohematology and Rheumatology, Necker Hospital Group, Paris, France.ORCID 0000-0002-0030-8094
Claire FieschiDepartment of Clinical Immunology, and Immunodeficiencies Center, Saint-Louis Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.ORCID 0000-0003-2136-6178
Samuel DeshayesDepartment of Internal Medicine, University Hospital of Caen, Caen, France.ORCID 0000-0001-8887-3233
Delphine GobertDepartment of Internal Medicine, University Hospital of St Antoine, Assistance Publique-Hôpitaux de Paris, Paris, France.
Clément GourguechonDepartment of Clinical Hematology and Cellular Therapy, University Hospital of Amiens, Amiens, France.ORCID 0000-0003-0740-2419
Hélène HeniqueDepartment of Internal Medicine, University Hospital of Tours, Tours, France.
Sebastien HumbertDepartment of Internal Medicine, University Hospital of Besançon, Besançon, France.ORCID 0000-0003-0454-0840
Carole LacoutDepartment of Internal Medicine, University Hospital of Angers, Angers, France.ORCID 0000-0003-2875-6048
Ronan Le CallochDepartment of Hematology Inpatient Care, Hospital of Quimper, Quimper, France.ORCID 0000-0001-5883-1825
Marc MichelDepartment of Internal Medicine, Henri Mondor University Hospital, and Center of Reference for Autoimmune Cytopenias in Adults, Assistance Publique-Hôpitaux de Paris, Créteil, France.
Marie-Lea Piel-JulianDepartment of Internal Medicine, University Hospital of Toulouse, Toulouse, France.ORCID 0000-0002-2619-0952
Jean François ViallardDepartment of Internal Medicine and Infectious Diseases, University Hospital of Bordeaux, Bordeaux, France.ORCID 0000-0001-7500-9323
Alain LescoatDepartment of Internal Medicine, University Hospital of Rennes, Rennes, France.
Bertrand GodeauDepartment of Internal Medicine, Henri Mondor University Hospital, and Center of Reference for Autoimmune Cytopenias in Adults, Assistance Publique-Hôpitaux de Paris, Créteil, France.ORCID 0000-0002-6023-5990
Antoinette PerlatDepartment of Internal Medicine, University Hospital of Rennes, Rennes, France.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

abstractPredominantly antibody deficiencies have an estimated prevalence of >1 in 25 000. Their classical phenotype entails the association of autoimmune manifestations with increased susceptibility to infections. Up to 8% of these patients ultimately develop immune thrombocytopenic purpura (ITP). Reducing the risk for infections and considering nonimmunosuppressive treatments, such as thrombopoietin receptor agonists (TPO-RAs), are important considerations for these patients. This nationwide retrospective case series assessed the outcomes and safety of TPO-RAs as treatment for ITP in adults diagnosed with predominantly antibody deficiencies. Response and complete response to treatment were defined as platelet count reaching 30 × 109/L and 100 × 109/L, respectively. We analyzed data from 28 patients. The median follow-up time after introduction of the first TPO-RAs was 33 months (range, 2 weeks to 10.6 years). After 6 weeks of follow-up, response was achieved in 24 of the 28 patients (85.7%), and among those, 21 patients (75%) displayed a complete response. At the last available follow-up visit, only 7 patients (25%) needed second-line therapies for ITP, and among those, only 5 patients (17.9%) received immunosuppressants. Only 3 patients (10.7%) reported laboratory-confirmed hepatobiliary adverse events of light or mild severity and 3 patients (10.7%) reported thrombotic events. In conclusion, TPO-RAs seemed to be an effective and safe option of treatment in these case series. Our results suggest that eltrombopag or romiplostim should be considered as second-line therapy for ITP related to predominantly antibody deficiencies.

Indexed as

Purpura, Thrombocytopenic, IdiopathicReceptors, ThrombopoietinAdultAgedAged, 80 and overBenzoatesFemaleHumansHydrazinesMaleMiddle AgedPyrazolesReceptors, FcRecombinant Fusion ProteinsRetrospective StudiesThrombopoietinBenzoateseltrombopagHydrazinesPyrazolesReceptors, FcReceptors, ThrombopoietinRecombinant Fusion ProteinsromiplostimThrombopoietin

Identifiers

PMID39348667
PMCPMC11696776

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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.