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Real-world Experience of Caplacizumab for Adolescents in the First-line Setting: Case Reports.
Tran, Thuy B; Boyce, Jennifer; Mitchell, Heather; Speaks, Jeron; Arnall, Justin R.
Affiliation
  • Tran TB; Department of Pharmacy, Specialty Pharmacy Services, Atrium Health, Charlotte, NC.
J Pediatr Hematol Oncol ; 45(8): e1031-e1034, 2023 11 01.
Article in En | MEDLINE | ID: mdl-37526365
Acquired thrombotic thrombocytopenic purpura (aTTP) is a thrombotic microangiopathy resulting in high mortality. Caplacizumab is approved for treatment of adults with acquired thrombotic thrombocytopenic purpura that has shown faster platelet normalization, clinical improvement, and reduced risk of recurrent/refractory disease. We report 2 cases of adolescents treated off-label with caplacizumab who were able to stop before 30 days from end of plasma exchange after platelets normalized and ADAMTS13 activity recovered to >20% to 30%. Our results show similar efficacy to other reports of patients under 18 receiving caplacizumab in first line, regardless of plasma exchange strategy, and may offer insight into early cessation criteria.
Subject(s)

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Purpura, Thrombotic Thrombocytopenic / Single-Domain Antibodies Limits: Adolescent / Adult / Humans Language: En Journal: J Pediatr Hematol Oncol Journal subject: HEMATOLOGIA / NEOPLASIAS / PEDIATRIA Year: 2023 Document type: Article Country of publication: United States

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Purpura, Thrombotic Thrombocytopenic / Single-Domain Antibodies Limits: Adolescent / Adult / Humans Language: En Journal: J Pediatr Hematol Oncol Journal subject: HEMATOLOGIA / NEOPLASIAS / PEDIATRIA Year: 2023 Document type: Article Country of publication: United States