Generic and disease-specific quality of life among youth and young men with Hemophilia in Canada

Abstract

BackgroundThis study was undertaken to explore the longitudinal patterns of health-related quality of life (HRQoL) among youth and young adults with Hemophilia A (HA) over a 3-year period. This report presents the baseline characteristics of the study cohort.MethodsMales, 14 to 29 years of age, with predominantly severe HA were recruited from six treatment centres in Canada. Subjects completed a comprehensive survey. HRQoL was measured using: the CHO-KLAT2.0 (youth), Haemo-QoL-A (young adults) and the SF-36v2 (all).Results13 youth (mean age = 15.7, range = 12.9-17.9 years) and 33 young adults (mean age = 23.6; range = 18.4 -28.7 years) with moderate (7 %) and severe (93 %) HA were enrolled. All were on a prophylactic regimen with antihemophilic factor (Helixate FS®) during the study. The youth had minimal joint damage (mean HJHS = 5.2) compared to young adults (mean HJHS = 13.3). The mean HRQoL scores for youth were: 79.2 (SD = 11.9) for the CHO-KLAT, and 53.0 (5.5) and 52.3 (6.8) for the SF-36 Physical Component Summary (PCS) and Mental Component Summary (MCS) scores respectively. The mean HRQoL scores for young adults were: 85.8 (9.5) for the Haemo-Qol-A, and 50.8 (6.4) and 50.9 (8.8) for PCS and MCS respectively. PCS and MCS scores were comparable to published Canadian norms, however significant differences were found for the domains of Physical Functioning and Bodily Pain. The disease-specific HRQoL scores were weakly correlated with the PCS for youth (CHO-KLAT vs. PCS r = 0.28, p = 0.35); and moderately correlated for the MCS (r = 0.39, p = 0.19). Haemo-QoL-A scores for young adults were strongly correlated with the PCS (r = 0.53, p = 0.001); and weakly correlated with the MCS (r = 0.26, p = 0.13). Joint status as assessed by HJHS was correlated with PCS scores. A history of lifelong prophylaxis resulted in better PCS but worse MCS scores.ConclusionDespite having hemophilia, the youth in this cohort have minimal joint disease and good HRQoL. The young adults demonstrated more joint disease and slightly worse HRQoL in the domains of physical functioning and pain. The data presented here provide new information to inform the selection of Health Related Quality of Life (HRQoL) instruments for use in future clinical trials involving persons with hemophilia.Trial registrationClinicalTrials.gov : NCT01034904. Study funded by CSL Behring Canada.Electronic supplementary materialThe online version of this article (doi:10.1186/s12878-016-0052-x) contains supplementary material, which is available to authorized users.

Adherence to treatment regimen and bleeding rates in a prospective cohort of youth and young adults on low-dose daily prophylaxis for severe hemophilia A

“…SF-36 also allows comparison with normal population or with groups with other diagnoses. In our cohort, the PCS and MCS summary scores of SF-36v2 varied minimally from baseline to the end of study and were similar to those reported at baseline in the larger CHeQoLS study (mean PCS 51.7 ± 6.5, mean MCS 51.1 ± 8.6) [7]. This low variability could be explained by the fact that our subjects were already on a LDDP regimen at study entry, but also by the low number of subjects enrolled in our study.…”

“…In addition, prophylaxis prevents bleeding and reduces the risk of life-threatening hemorrhages [10, 11, 18]. The purpose of our study was to describe clinical outcomes (bleeding rates and joint health), adherence to treatment and HRQoL in a cohort of patients treated on a LDDP regimen, while participating in a non-interventional and longitudinal study of youth and young adults with moderate or severe HA in Canada [7]. …”

“…CHeQoLS was a prospective non-interventional study describing longitudinal HRQoL in 48 youth and young adults with moderate or severe HA [7], conducted over three years in 6 Canadian Hemophilia Treatment Centres (HTCs). Male subjects 14 to 29 years of age were eligible for CHeQoLS if they had moderate (FVIII level 0.02 – 0.05 U/ml) or severe HA (FVIII level <0.02 U/ml) and were using the antihemophilic factor concentrate Helixate FS ® (CSL Behring, King of Prussia, PA, USA) as a prophylactic or on-demand treatment.…”

“…Male subjects 14 to 29 years of age were eligible for CHeQoLS if they had moderate (FVIII level 0.02 – 0.05 U/ml) or severe HA (FVIII level <0.02 U/ml) and were using the antihemophilic factor concentrate Helixate FS ® (CSL Behring, King of Prussia, PA, USA) as a prophylactic or on-demand treatment. Exclusion criteria were presence of a current inhibitor to FVIII (defined as ≥ 0.6 Bethesda Units/mL), human immunodeficiency virus (HIV) or symptomatic hepatitis C virus (HCV) infection [7]. CHeQoLS was funded by CSL Behring Canada and was registered in Clinical Trials.gov under the registry number NCT01034904 on December 17, 2009.…”

Patient powered prophylaxis: A 12‐month study of individualized prophylaxis in adults with severe haemophilia A

“…First, we aimed to identify motivators to support patients' intrinsic desire to change their prophylaxis regimens, making increased infusion frequency less likely to be burdensome. Second, lack of difference in HRQoL in other studies may be due to small numbers, as the global HRQoL score is recognized to be insensitive to changes in patients with severe haemophilia compared to physical functioning or bodily pain subscales . Third, our patient population had a higher ABR and higher prevalence of joint disease at baseline compared to previous studies of daily prophylaxis; hence, they are more likely to derive a benefit in bleed reduction and improved HRQoL.…”