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Long-term use of the thrombopoietin-mimetic romiplostim in children with severe chronic immune thrombocytopenia (ITP).
Pediatr Blood Cancer. 2015 02; 62(2):208-213.PB

Abstract

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 × 109 /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 × 109 /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 × 109 /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 × 109 /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 × 109 /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.

Authors+Show Affiliations

Department of Pediatrics, Division of Hematology, Weill Medical College of Cornell University, New York, New York.Children's Hospital of Orange County/University of California, Irvine, California.Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, Texas.Arkansas Children's Hospital, Little Rock, Arkansas.Children's Mercy Hospital, Kansas City, Missouri.Children's Hospital and Medical Center, University of Nebraska Medical Center, Omaha, Nebraska.Department of Pediatrics, Monroe Carell Jr. Children's Hospital at Vanderbilt, Nashville, Tennessee.Amgen Inc., Thousand Oaks, California.Amgen Inc., Thousand Oaks, California.

Pub Type(s)

Clinical Trial, Phase I
Clinical Trial, Phase II
Journal Article

Language

eng

PubMed ID

25345874

Citation

Bussel, James B., et al. "Long-term Use of the Thrombopoietin-mimetic Romiplostim in Children With Severe Chronic Immune Thrombocytopenia (ITP)." Pediatric Blood & Cancer, vol. 62, no. 2, 2015, pp. 208-213.
Bussel JB, Hsieh L, Buchanan GR, et al. Long-term use of the thrombopoietin-mimetic romiplostim in children with severe chronic immune thrombocytopenia (ITP). Pediatr Blood Cancer. 2015;62(2):208-213.
Bussel, J. B., Hsieh, L., Buchanan, G. R., Stine, K., Kalpatthi, R., Gnarra, D. J., Ho, R. H., Nie, K., & Eisen, M. (2015). Long-term use of the thrombopoietin-mimetic romiplostim in children with severe chronic immune thrombocytopenia (ITP). Pediatric Blood & Cancer, 62(2), 208-213. https://doi.org/10.1002/pbc.25136
Bussel JB, et al. Long-term Use of the Thrombopoietin-mimetic Romiplostim in Children With Severe Chronic Immune Thrombocytopenia (ITP). Pediatr Blood Cancer. 2015;62(2):208-213. PubMed PMID: 25345874.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Long-term use of the thrombopoietin-mimetic romiplostim in children with severe chronic immune thrombocytopenia (ITP). AU - Bussel,James B, AU - Hsieh,Loan, AU - Buchanan,George R, AU - Stine,Kimo, AU - Kalpatthi,Ram, AU - Gnarra,David J, AU - Ho,Richard H, AU - Nie,Kun, AU - Eisen,Melissa, Y1 - 2014/10/24/ PY - 2013/10/29/received PY - 2014/05/14/accepted PY - 2015/2/1/pubmed PY - 2015/2/1/medline PY - 2014/10/28/entrez KW - autoimmunity KW - bleeding KW - platelets KW - thrombopoietin SP - 208 EP - 213 JF - Pediatric blood & cancer JO - Pediatr Blood Cancer VL - 62 IS - 2 N2 - 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 × 109 /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 × 109 /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 × 109 /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 × 109 /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 × 109 /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. SN - 1545-5017 UR - https://www.unboundmedicine.com/medline/citation/25345874/Long_term_use_of_the_thrombopoietin_mimetic_romiplostim_in_children_with_severe_chronic_immune_thrombocytopenia__ITP__ L2 - https://doi.org/10.1002/pbc.25136 DB - PRIME DP - Unbound Medicine ER -