PMID- 25345874
OWN - NLM
STAT- MEDLINE
DCOM- 20171023
LR  - 20211021
IS  - 1545-5017 (Electronic)
IS  - 1545-5009 (Print)
IS  - 1545-5009 (Linking)
VI  - 62
IP  - 2
DP  - 2015 Feb
TI  - Long-term use of the thrombopoietin-mimetic romiplostim in children with severe 
      chronic immune thrombocytopenia (ITP).
PG  - 208-213
LID - 10.1002/pbc.25136 [doi]
AB  - 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 x 10(9) /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 x 10(9) /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 microg/kg/week to maintain platelet counts (50-200 x 10(9) /L, maximum 
      dose 10 microg/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 x 10(9) /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 microg/kg (Q1, Q3: 4.3, 8.0 microg/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 x 10(9) /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. (c) 2014. The Authors. Pediatr 
      Blood & Cancer published by Wiley Periodicals, Inc.
CI  - (c) 2014 The Authors. Pediatric Blood & Cancer published by Wiley Periodicals, Inc.
FAU - Bussel, James B
AU  - Bussel JB
AD  - Department of Pediatrics, Division of Hematology, Weill Medical College of 
      Cornell University, New York, New York.
FAU - Hsieh, Loan
AU  - Hsieh L
AD  - Children's Hospital of Orange County/University of California, Irvine, 
      California.
FAU - Buchanan, George R
AU  - Buchanan GR
AD  - Department of Pediatrics, University of Texas Southwestern Medical Center, 
      Dallas, Texas.
FAU - Stine, Kimo
AU  - Stine K
AD  - Arkansas Children's Hospital, Little Rock, Arkansas.
FAU - Kalpatthi, Ram
AU  - Kalpatthi R
AD  - Children's Mercy Hospital, Kansas City, Missouri.
FAU - Gnarra, David J
AU  - Gnarra DJ
AD  - Children's Hospital and Medical Center, University of Nebraska Medical Center, 
      Omaha, Nebraska.
FAU - Ho, Richard H
AU  - Ho RH
AD  - Department of Pediatrics, Monroe Carell Jr. Children's Hospital at Vanderbilt, 
      Nashville, Tennessee.
FAU - Nie, Kun
AU  - Nie K
AD  - Amgen Inc., Thousand Oaks, California.
FAU - Eisen, Melissa
AU  - Eisen M
AD  - Amgen Inc., Thousand Oaks, California.
LA  - eng
PT  - Clinical Trial, Phase I
PT  - Clinical Trial, Phase II
PT  - Journal Article
DEP - 20141024
PL  - United States
TA  - Pediatr Blood Cancer
JT  - Pediatric blood & cancer
JID - 101186624
RN  - 0 (Receptors, Fc)
RN  - 0 (Receptors, Thrombopoietin)
RN  - 0 (Recombinant Fusion Proteins)
RN  - 9014-42-0 (Thrombopoietin)
RN  - GN5XU2DXKV (romiplostim)
SB  - IM
MH  - Adolescent
MH  - Blood Platelets/*drug effects
MH  - Child
MH  - Child, Preschool
MH  - Chronic Disease
MH  - Female
MH  - Humans
MH  - Infant
MH  - Longitudinal Studies
MH  - Male
MH  - Platelet Count
MH  - Purpura, Thrombocytopenic, Idiopathic/*drug therapy
MH  - Receptors, Fc/*therapeutic use
MH  - Receptors, Thrombopoietin/*agonists
MH  - Recombinant Fusion Proteins/adverse effects/*therapeutic use
MH  - Thrombopoietin/adverse effects/*therapeutic use
MH  - Treatment Outcome
PMC - PMC4309514
OTO - NOTNLM
OT  - autoimmunity
OT  - bleeding
OT  - platelets
OT  - thrombopoietin
EDAT- 2015/02/01 00:00
MHDA- 2015/02/01 00:01
CRDT- 2014/10/28 06:00
PHST- 2013/10/29 00:00 [received]
PHST- 2014/05/14 00:00 [accepted]
PHST- 2015/02/01 00:00 [pubmed]
PHST- 2015/02/01 00:01 [medline]
PHST- 2014/10/28 06:00 [entrez]
AID - 10.1002/pbc.25136 [doi]
PST - ppublish
SO  - Pediatr Blood Cancer. 2015 Feb;62(2):208-213. doi: 10.1002/pbc.25136. Epub 2014 
      Oct 24.