Efficacy and safety of TPO receptor agonists in treatment of ITP associated with predominantly antibody deficiencies
Predominantly 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.
Authors
- M.L. Piel-Julian (ORCID: https://orcid.org/0000-0002-2619-0952)
- Claire Fieschi (ORCID: https://orcid.org/0000-0003-2136-6178)
- Lionel Galicier (ORCID: https://orcid.org/0000-0002-0360-7620)
- Ronan Le Calloch (ORCID: https://orcid.org/0000-0001-5883-1825)
- Antoinette Perlat (ORCID: https://orcid.org/0000-0002-4985-423X)
- Delphine Gobert
- Samuel Deshayes (ORCID: https://orcid.org/0000-0001-8887-3233)
- Marc Michel (ORCID: https://orcid.org/0000-0002-9822-7400)
- Jean‐François Viallard (ORCID: https://orcid.org/0000-0001-7500-9323)
- Nizar Mahlaoui (ORCID: https://orcid.org/0000-0002-0030-8094)
- Carole Lacout (ORCID: https://orcid.org/0000-0003-2875-6048)
- H. Henique
- Bertrand Godeau (ORCID: https://orcid.org/0000-0002-6023-5990)
- C. Gourguechon (ORCID: https://orcid.org/0000-0003-0740-2419)
- S. Humbert (ORCID: https://orcid.org/0000-0003-0454-0840)
- Alain Lescoat (ORCID: https://orcid.org/0000-0001-5289-3312)
- M. Soulard
Institutions
- Université de Tours (FR)
- Université Toulouse III - Paul Sabatier (FR)
- Université de Bordeaux (FR)
- Université Fédérale de Toulouse Midi-Pyrénées (FR)
- Université Paris-Est Créteil (FR)
- Université Paris Cité (FR)
- Hereditary Disease Foundation (US)
- Université Rennes 2 (FR)
- Sorbonne Université (FR)
- Centre Hospitalier Universitaire de Besançon (FR)
- Sorbonne Paris Cité (FR)
- Centre Hospitalier Universitaire de Tours (FR)
- Centre Hospitalier Universitaire Amiens-Picardie (FR)
- Assistance Publique – Hôpitaux de Paris (FR)
- Toulouse Mathematics Institute (FR)
- Hôpital Saint-Antoine (FR)
- Bordeaux Population Health (FR)
- Hôpital Saint-André (FR)
- Centre Hospitalier Universitaire de Caen Normandie (FR)
- Centre Hospitalier de Cornouaille (FR)
- Hôpital Cardiologique du Haut-Lévêque (FR)
- Hôpitaux Universitaires Henri-Mondor (FR)
- Hôpital Saint-Louis (FR)
- Centre Hospitalier Universitaire de Rennes (FR)
- Center for Rheumatology (US)
- Hôpital de la Timone (FR)
- Université d'Angers (FR)
- Université de Rennes (FR)
- Université de Caen Normandie (FR)
Publication Details
- Journal
- Blood Advances
- Published
- 2024-09-30
- DOI
- https://doi.org/10.1182/bloodadvances.2024014370
- Citations
- 5
- Primary Topic
- Platelet Disorders and Treatments
- Type
- article
- Field-Weighted Citation Impact
- 2.27