Adolescents and young adults with newly diagnosed primary immune thrombocytopenia

Haematologica. 2023 Oct 1;108(10):2783-2793. doi: 10.3324/haematol.2022.282524.

Abstract

Current immune thrombocytopenia (ITP) guidelines target children and adults, leading to oversimplification. Adolescents and young adults (AYAS) comprise a separate group with distinct health and psychosocial issues. This study aimed to describe the clinical presentation and therapeutic strategies of ITP among AYAS. We analyzed data from two large ITP registries (PARC-ITP; CARMEN-France) and included newly diagnosed ITP patients (aged 12-25 years) with an initial platelet counts of <100×109/L. Patients with secondary ITP or non-immune thrombocytopenia (n=57) and pregnant women (n=10) were excluded. Of the 656 cases of AYAS with primary ITP registered from 2004 up to 2021, 12-month follow-up data were available for 72%. The initial median platelet count was 12×109/L. In 109 patients (17%), the diagnosis was incidental, without documented bleeding. Apart from gynecological bleeding, the clinical and therapeutical characteristics of females and males were similar. Platelet-enhancing drugs were reported in 66%, 45%, and 30% of patients at diagnosis, 1-6 months, and 6-12 months after diagnosis, respectively. Corticosteroids were the preferred treatment at all time points. At 12 months, 50% of all patients developed chronic ITP. In the subgroup of patients with initial severe thrombocytopenia (<20×109/L), those receiving frontline treatment had a higher remission rate at 1 year than those who followed an initial watch-and-wait strategy (53% and 32%; P<0.05). Our analysis indicates that the remission rate at 1 year may be associated with the initial treatment strategy. This hypothesis must be confirmed in prospective studies.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Child
  • Female
  • Hemorrhage / diagnosis
  • Humans
  • Male
  • Platelet Count
  • Pregnancy
  • Prospective Studies
  • Purpura, Thrombocytopenic, Idiopathic* / drug therapy
  • Purpura, Thrombocytopenic, Idiopathic* / therapy
  • Thrombocytopenia*
  • Young Adult

Grants and funding

Funding: Funding was receieved from the Platelet Disease Support Association (PDSA), Ohio, USA; Stiftung hämatologische Forschung, Basel, CH; Stiftung zur Förderung medizinischer und biologischer Forschung, Arlesheim, CH. The PARC registry received financial support from the Intercontinental Cooperative ITP Study Group (ICIS). The CARMEN registry received support from the French National Society of Internal Medicine, Toulouse Referral Center for Autoimmune Cytopenias, Toulouse University Hospital, Amgen, CSL Behring, Grifols, and Novartis.