Pilot study of the effect of romiplostim on child health-related quality of life (HRQoL) and parental burden in immune thrombocytopenia (ITP)

Pediatric Blood and Cancer

Volumn 58, Issue 3, 2012, Pages 395-398

  Klaassen, Robert J ^a   Mathias, Susan D ^b   Buchanan, George ^c   Bussel, James ^d   Deuson, Robert ^e   Young, Nancy L ^f   Collier, Anderson ^g   Bomgaars, Lisa ^h   Blanchette, Victor ⁱ  

^a CHILDREN S HOSPITAL OF EASTERN ONTARIO   (Canada)

^b Health Outcomes Solutions   (United States)

^c UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER   (United States)

^d WEILL CORNELL MEDICAL COLLEGE   (United States)

^e AMGEN INC   (United States)

^f LAURENTIAN UNIVERSITY   (Canada)

^g LEHIGH VALLEY HOSPITAL   (United States)

^h BAYLOR COLLEGE OF MEDICINE   (United States)

ⁱ HOSPITAL FOR SICK CHILDREN   (Canada)

Author keywords

Idiopathic; Parents; Purpura; Quality of life; Questionnaires; Thrombocytopenic

Indexed keywords

PLACEBO; ROMIPLOSTIM;

ADOLESCENT; ADULT; ARTICLE; CAREGIVER BURDEN; CHILD; CLINICAL ARTICLE; CONTROLLED STUDY; DOUBLE BLIND PROCEDURE; DRUG EFFECT; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; FAMILY; FEMALE; HUMAN; IDIOPATHIC THROMBOCYTOPENIC PURPURA; IMMUNE THROMBOCYTOPENIA; MALE; MULTICENTER STUDY; PARENT; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PILOT STUDY; PRIORITY JOURNAL; QUALITY OF LIFE; QUESTIONNAIRE; RANDOMIZED CONTROLLED TRIAL; SCORING SYSTEM; SELF REPORT; TEST RETEST RELIABILITY; THROMBOCYTE COUNT; THROMBOCYTOPENIA; TREATMENT OUTCOME;

ADOLESCENT; CASE-CONTROL STUDIES; CHILD; CHILD, PRESCHOOL; COST OF ILLNESS; FEMALE; HUMANS; INFANT; MALE; PARENTS; PURPURA, THROMBOCYTOPENIC, IDIOPATHIC; QUALITY OF LIFE; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RECEPTORS, FC; RECOMBINANT FUSION PROTEINS; THROMBOPOIETIN;

EID: 84855770524     PISSN: 15455009     EISSN: 15455017     Source Type: Journal    
DOI: 10.1002/pbc.23312     Document Type: Article

References (18)

1. Nugent D, McMillan R, Nichol JL, et al. Pathogenesis of chronic immune thrombocytopenia: Increased platelet destruction and/or decreased platelet production. Br J Haematol 2009; 146: 585-596.
2. Yong M, Schoonen WM, Li L, et al. Epidemiology of paediatric immune thrombocytopenia in the General Practice Research Database. Br J Haematol 2010; 149: 855-864.
3. Buchanan GR. Thrombocytopenia during childhood: What the pediatrician needs to know. Pediatr Rev 2005; 26: 401-409.
4. Kuhne T, Buchanan GR, Zimmerman S, et al. A prospective comparative study of 2540 infants and children with newly diagnosed idiopathic thrombocytopenic purpura (ITP) from the Intercontinental Childhood ITP Study Group. J Pediatr 2003; 143: 605-608.
5. Kuhne T, Imbach P, Bolton-Maggs PH, et al. Newly diagnosed idiopathic thrombocytopenic purpura in childhood: An observational study. Lancet 2001; 358: 2122-2125.
6. Bussel JB, Kuter DJ, George JN, et al. AMG 531, a thrombopoiesis-stimulating protein, for chronic ITP. N Engl J Med 2006; 355: 1672-1681.
7. Bussel JB, Kuter DJ, Pullarkat V, et al. Safety and efficacy of long-term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Blood 2009; 113: 2161-2171.
8. Kuter DJ, Bussel JB, Lyons RM, et al. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: A double-blind randomised controlled trial. Lancet 2008; 371: 395-403.
9. Newland A, Caulier MT, Kappers-Klunne M, et al. An open-label, unit dose-finding study of AMG 531, a novel thrombopoiesis-stimulating peptibody, in patients with immune thrombocytopenic purpura. Br J Haematol 2006; 135: 547-553.
10. Bussel JB, Buchanan GR, Nugent DJ, et al. A randomized, double-blind study of romiplostim to determine its safety and efficacy in children with immune thrombocytopenia. Blood 2011; 118: 28-36.
11. Klaassen R. Acute immune thrombocytopenic purpura: What do we do when things go well? Paediatr Child Health 2002; 7: 384-385.
12. Barnard D, Woloski M, Feeny D, et al. Development of disease-specific health-related quality-of-life instruments for children with immune thrombocytopenic purpura and their parents. J Pediatr Hematol Oncol 2003; 25: 56-62.
13. Klaassen RJ, Blanchette VS, Barnard D, et al. Validity, reliability, and responsiveness of a new measure of health-related quality of life in children with immune thrombocytopenic purpura: The Kids' ITP Tools. J Pediatr 2007; 150: 510-515 5 e1.
14. George JN, Woolf SH, Raskob GE, et al. Idiopathic thrombocytopenic purpura: A practice guideline developed by explicit methods for the American Society of Hematology. Blood 1996; 88: 3-40.
15. Aaronson N, Alonso J, Burnam A, et al. Assessing health status and quality-of-life instruments: Attributes and review criteria. Qual Life Res 2002; 11: 193-205.
16. Tamary HRJ, Chitlur M, Nugent DJ. Consensus Paper-ICIS Expert Meeting Basel 2009 treatment milestones in immune thrombocytopenia. Ann Hematol 2010; 89: S5-S10.
17. George JN, Mathias SD, Go RS, et al. Improved quality of life for romiplostim-treated patients with chronic immune thrombocytopenic purpura: Results from two randomized, placebo-controlled trials. Br J Haematol 2009; 144: 409-415.
18. Cheng G, Saleh MN, Marcher C, et al. Eltrombopag for management of chronic immune thrombocytopenia (RAISE): A 6-month, randomised, phase 3 study. Lancet 2011; 377: 393-402.