Author(s)
Curtis R1, Ding Y2, Aliyev ER2, Wu J2, Koerper MA3, Lou M2, Ullman MM4, Tran Jr. DQ5, Baker J6, Riske B7, Nichol MB2
1Factor VIII Computing, Berkeley, CA, USA, 2University of Southern California, Los Angeles, CA, USA, 3University of California San Francisco, San Francisco, CA, USA, 4Gulf States Hemophilia & Thrombophilia Center, University of Texas health Science Center at Houston, Houston, TX, Houston, TX, USA, 5Emory University, Hemophilia of Georgia Center for Bleeding & Clotting Disorders of Emory, Atlanta, GA, USA, 6The Center for Comprehensive Care & Diagnosis of Inherited Blood Disorders and University of California Los Angeles, Orange, CA, USA, 7University of Colorado Denver, Aurora, CO, USA
OBJECTIVES: We compared participant characteristics between the Hemophilia Utilization Group Studies Part Va (HUGS-Va) and a long-term follow-up study (HUGS-LTS) to investigate the impact of changes of participants’ characteristics on annualized bleeding rates (ABR) in persons with hemophilia A, a genetic bleeding disorder. METHODS: Self-reported bleeds were obtained from periodic HUGS-Va surveys conducted 2005-2007, and a 6-month recall survey from HUGS-LTS in 2014. Clotting factor dispensing records were collected prospectively for two years in HUGS-Va, and retrospectively for six months prior to HUGS-LTS. ABR and annualized factor dispensing (unit/kg body weight) were calculated and compared by age (children 2-11 years, adolescents 12-20 years, and adults ≥21 years) using ANOVA, and between the studies using paired T-tests. RESULTS: Sixty-nine participants completed both studies. Participants or parents of children participants had a higher rate of college education (76.5% vs. 98.5%) and full-year health insurance (88.4% vs. 95.7%) in HUGS-Va than in the HUGS-LTS. Adolescents and adults had an increased prophylactic treatment rate (60.0% to 78.6% and 30.8% to 69.2%, respectively) from HUGS-Va to HUGS-LTS, all P<0.05. Children had the highest mean/median factor dispensing (4822±4152/3300, 5061±3764/5336 IU/kg/year) and lowest mean/median ABR (3.9±4.4/2.8, 4.9±5.3/4.0); while adults had the lowest mean/median factor dispensing (2084±1870/1582, 4612±2571/4290 IU/kg/year) and highest ABR (12.4±9.2/14.0, 13.3±21.5/6.0) in both HUGS-Va and HUGS-LTS, respectively. Overall 70% of participants increased factor dispensing, while 51% of participants increased ABR. However, the mean/median ARB was not significant different between HUGS-Va (7.4±8.6/3.8) and HUGS-LTS (8.7±14.7/4.0), P=0.45. Participants who used episodic treatment had higher ABR (15.7) than those who used prophylactic treatment (10), P=0.41 in HUGS-LTS. CONCLUSIONS: Children and adolescents with higher factor dispensing showed lower ABR than adults, mean ABR had not significant changed while aging. Increased in rates of college educational and health insurance appear to increase use of prophylactic treatment and factor dispensing, which lowers ABR.
Conference/Value in Health Info
2019-05, ISPOR 2019, New Orleans, LA, USA
Value in Health, Volume 22, Issue S1 (2019 May)
Code
PRO53
Topic
Clinical Outcomes, Health Service Delivery & Process of Care, Patient-Centered Research
Topic Subcategory
Clinical Outcomes Assessment, Patient-reported Outcomes & Quality of Life Outcomes, Treatment Patterns and Guidelines
Disease
Rare and Orphan Diseases
