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Blind men and the refractory ITP elephant.
Chaturvedi S, McCrae KR
(2016) Blood 128: 1537-8
MeSH Terms: Humans, Inosine Triphosphate, Purpura, Thrombocytopenic, Idiopathic
Added October 23, 2016
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3 MeSH Terms
Treatment of chronic immune thrombocytopenia in children with romiplostim.
Chaturvedi S, McCrae KR
(2016) Lancet 388: 4-6
MeSH Terms: Female, Humans, Male, Purpura, Thrombocytopenic, Idiopathic, Receptors, Fc, Receptors, Thrombopoietin, Recombinant Fusion Proteins, Thrombopoietin
Added April 25, 2016
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Long-term use of the thrombopoietin-mimetic romiplostim in children with severe chronic immune thrombocytopenia (ITP).
Bussel JB, Hsieh L, Buchanan GR, Stine K, Kalpatthi R, Gnarra DJ, Ho RH, Nie K, Eisen M
(2015) Pediatr Blood Cancer 62: 208-213
MeSH Terms: Adolescent, Blood Platelets, Child, Child, Preschool, Chronic Disease, Female, Humans, Infant, Longitudinal Studies, Male, Platelet Count, Purpura, Thrombocytopenic, Idiopathic, Receptors, Fc, Receptors, Thrombopoietin, Recombinant Fusion Proteins, Thrombopoietin, Treatment Outcome
Show Abstract · Added January 21, 2015
BACKGROUND - Treatment of chronic severe pediatric ITP is not well studied. In a phase 1/2 12-16-week study, 15/17 romiplostim-treated patients achieved platelet counts ≥50 × 10 /L, and romiplostim treatment was well tolerated. In a subsequent open-label extension (≤109 weeks), 20/22 patients received romiplostim; all achieved platelet counts >50 × 10 /L. Twelve patients continued in a second extension (≤127 weeks). Longitudinal data from start of romiplostim treatment through the two extensions were evaluated to investigate the safety and efficacy of long-term romiplostim treatment in chronic severe pediatric ITP.
PROCEDURE - Patients received weekly subcutaneous romiplostim, adjusted by 1 µg/kg/week to maintain platelet counts (50-200 × 10 /L, maximum dose 10 µg/kg). Bone marrow examinations were not required.
RESULTS - At baseline, patients were median age 10.0 years; median ITP duration 2.4 years; median platelet count 13 × 10 /L; 73% were male; and 36% had prior splenectomy. Median romiplostim treatment duration was 167 weeks (Q1, Q3: 78,227 weeks), and median average weekly dose was 5.4 µg/kg (Q1, Q3: 4.3, 8.0 µg/kg). Seven patients discontinued treatment: four withdrew consent, two were noncompliant, and one received alternative therapy. None withdrew because of adverse events (AEs). After the first 12 weeks, median platelet counts remained >50 × 10 /L. Eight (36.4%) patients received rescue medication, and 14 (63.6%) used concurrent ITP therapy. Seven patients (31.8%) reported serious AEs, and two (9.1%) reported life-threatening AEs (both thrombocytopenia); there were no serious AEs attributed to treatment and no fatalities.
CONCLUSIONS - Long-term romiplostim treatment in this small cohort increased and maintained platelet counts for over 4 years in children with ITP with good tolerability and without significant toxicity. Pediatr Blood Cancer 2015;62:208-213. © 2014. The Authors. Pediatr Blood & Cancer published by Wiley Periodicals, Inc.
© 2014 The Authors. Pediatric Blood & Cancer published by Wiley Periodicals, Inc.
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17 MeSH Terms