Details

Autor(en) / Beteiligte

• Kalinina, Irina I
• Antonova, Khristina S
• Avdonin, Pavel V
• Klebanova, Elizaveta E
• Kotskaya, Natalia N
• Kurnikova, Elena E
• Shutova, Alexandra D
• Matveev, Victor E
• Maschan, Alexey A

Titel

Successful Treatment of Acquired Thrombotic Thrombocytopenic Purpura With Caplacizumab Combined With Plasma Exchanges and Immune Suppression in 3 Children

Ist Teil von

• Journal of pediatric hematology/oncology, 2024-04, Vol.46 (3), p.e220

Ort / Verlag

United States

Erscheinungsjahr

2024

Link zum Volltext

Quelle

MEDLINE

Beschreibungen/Notizen

• Acquired thrombotic thrombocytopenic (aTTP) purpura is a life-threatening condition that can lead to devastating thromboembolic events. Recently, caplacizumab has been shown to rapidly restore platelet numbers and reduce the risk of severe end-organ damage when added to plasma exchanges (PEXs) and immunosuppression (IST). Here, we report the outcomes in 3 children with aTTP who were treated with caplacizumab in combination with PEXs and IST. In all 3 patients, platelet count increased to >15,000/mm 3 in 24 h and normalized on day 4, whereas normalization of ADAMTS13 activity >50% and elimination of the inhibitor was achieved after 18 to 89 days. Epistaxis was observed in 2 patients and was the only side effect related to caplacizumab. Caplacizumab is a promising agent for first-line treatment of children with aTTP.