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. 2024 Dec 24;8(24):6171-6182.
doi: 10.1182/bloodadvances.2024014370.

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

Margaux Soulard  1 , Lionel Galicier  2 , Nizar Mahlaoui  3   4 , Claire Fieschi  2 , Samuel Deshayes  5 , Delphine Gobert  6 , Clément Gourguechon  7 , Hélène Henique  8 , Sebastien Humbert  9 , Carole Lacout  10 , Ronan Le Calloch  11 , Marc Michel  12 , Marie-Lea Piel-Julian  13 , Jean François Viallard  14 , Alain Lescoat  1 , Bertrand Godeau  12 , Antoinette Perlat  1
Affiliations

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

Margaux Soulard et al. Blood Adv. .

Abstract

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.

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Conflict of interest statement

Conflict-of-interest disclosure: B.G. reports serving as an expert for Amgen, Grifols, and Novartis. H.H. reports serving as an expert for Amgen and Novartis. L.G. reports serving as an expert for Amgen, GlaxoSmithKline, Novartis, and Sanofi. M.M. reports serving as a consultant (advisory boards) for and receiving speaker fees from Grifols, Novartis, Sanofi, and Sobi. The remaining authors declare no competing financial interests.

Figures

None
Graphical abstract
Figure 1.
Figure 1.
Flow chart: study population. ALPS, autoimmune lymphoproliferative syndrome; CeReCaI, Center of Reference for Autoimmune Cytopenias in Adults; CEREDIH, Center of Reference for Hereditary Immunodeficiency Disorders; Di-Georges, Di-Georges syndrome; PC, platelet count; WA, Wiskott-Aldrich syndrome.
Figure 2.
Figure 2.
Swimmer plot. The x-axis represents time since the introduction of TPO-RAs, measured in months and the y-axis shows each of the 28 patient’s individual treatment courses. Colors are used to indicate different responses: the gray color denotes absence of response or insufficient response (with the use of immunosuppressants for an ITP relapse); the green color indicates a response or complete response to TPO-RAs, with the treatment ongoing; and the green/grey color represents a response or complete response to TPO-RAs, after discontinuation of treatment. Hourglass symbols: orange, immunosuppressants used concomitantly to TPO-RAs; red, time at which immunosuppressants were introduced for an ITP relapse; and purple, time at which immunosuppressants were introduced for other medical reasons. Lines represent time when TPO-RAs were discontinued.
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