We found a match
Your institution may have access to this item. Find your institution then sign in to continue.
- Title
SINGLE-CENTRE REAL-LIFE EXPERIENCE ON THE USE OF THROMBOPOIETIN RECEPTOR AGONIST (TPO-RA) FOR THE MANAGEMENT OF IMMUNE THROMBOCYTOPENIA IN CHILDREN.
- Authors
OBRISCA, RADU; COLITA, ANCA; SERBANICA, ANDREEA; BICA, ANA MARIA; JERCAN, CRISTINA; MARCU, ANDRA DANIELA; RADU, LETITIA ELENA; MARCU, ANA MARIA; COLITA, ANDREI; ORBAN, CARMEN
- Abstract
Thrombopoietin receptor agonists (TPO-RA) represent a therapeutic option for paediatric patients with chronic immune thrombocytopenia (ITP) not responding to other treatments. However, the decision to use TPO-RA in paediatric cases is made on a case-by-case basis, and the potential benefits and risks are carefully considered by the treating physician. This is a retrospective unicentric study conducted in the Department of Paediatrics, Fundeni Clinical Institute, Bucharest, Romania. We assessed the efficacy and safety of TPO-RAs during the first year of treatment in patients 1 - 18 years with chronic ITP. We collected demographic data (age, previous therapies), TPO-RA data (dose, monitoring, side effects), and outcome data (platelet count, additional treatments) during the study period 2017 - 2022. We included 33 patients with chronic ITP of which 24 patients received eltrombopag and 9 romiplostin. The median age at ITP diagnosis was 7 years and 10 years at TPO-RA initiation. The median platelet count at ITP diagnosis and TPO-RA start was 30 x 109 per L. Overall 93.9% of patients achieved a platelet count over 50 x 109 per L, with a median response time of 5 weeks. One-third of patients required additional therapies. The treatment discontinuation rate was 6% due to stable treatment response and 6% due to lack of response. Adverse effects (AE) were presented in 30% of patients. There were no significant differences in response between the two products. Both products provide a safe and efficient therapeutic option for paediatric patients with chronic ITP. However, most patients require long-term treatment implicating high costs and potential increase of AE, with efficiency and safety not evaluated in large studies.
- Subjects
BUCHAREST (Romania); THROMBOPOIETIN receptor agonists; IDIOPATHIC thrombocytopenic purpura; CHILD patients; BLOOD platelet disorders; TERMINATION of treatment; PLATELET count; CHRONICALLY ill
- Publication
Farmacia, 2024, Vol 72, Issue 1, p185
- ISSN
0014-8237
- Publication type
Article
- DOI
10.31925/farmacia.2024.1.20