A prospective study of health‐related quality of life of boys with severe haemophilia A in China: comparing on‐demand to prophylaxis treatment

Wu, Runhui; Sun, Jing; Liu, Y.; Xue, Feng; Wang, H.; Tang, Ling; Zhao, Yongqiang; Li, K.; Yang, Renchi; Hu, Yu; Luke, Koon Hung; Poon, Man‐Chiu; Blanchette, Victor S.; Usuba, Koyo; Young, Nancy L.

doi:10.1111/hae.13198

Cited by 19 publications

(34 citation statements)

References 25 publications

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“…The generic measure of activity, the ASK, also provides supplementary information that enables the children in Jamaica to be compared with other studies. The reported mean score of 82.0 for Jamaican children in this study was slightly higher (better) than the mean from a Chinese sample with severe haemophilia (mean = 74.9 during on‐demand therapy) but lower (worse) than a Brazilian sample (median = 92.0) in which 70% of children had severe haemophilia . These data are consistent with the availability of clotting factor concentrates in the countries referenced.…”

Section: Discussionsupporting

confidence: 83%

“…23 The generic measure of activity, the ASK, also provides supplementary information that enables the children in Jamaica to be compared with other studies. The reported mean score of 82.0 for Jamaican children in this study was slightly higher (better) than the mean from a Chinese sample with severe haemophilia (mean = 74.9 during on-demand therapy) 24 and 77.0 in European countries 27 ; and mean Haemo-QoL-A score of 85.6 in the USA. 28 This indicates that there is a large and significant gap in HRQoL between PWH in countries with different access to clotting factor concentrates.…”

Section: Discussioncontrasting

confidence: 78%

“… Target joint: A target joint is defined as one in which at least 3 or 4 bleeds have occurred within a 3‐ to 6‐mo period. A joint is no longer considered a target joint when there has been no bleeding into that joint for 12 mo …”

Section: Resultsmentioning

confidence: 99%

“…A joint is no longer considered a target joint when there has been no bleeding into that joint for 12 mo. 24 included 8 child-parent dyads. The child self-and parent-reported scores were similar: means of 60.2 and 58.0, respectively.…”

Section: Health-related Quality Of Lifementioning

confidence: 99%

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The Jamaican Haemophilia Registry: Describing the burden of disease

et al. 2018

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Section: Discussionsupporting

confidence: 83%

Section: Discussioncontrasting

confidence: 78%

Section: Resultsmentioning

confidence: 99%

Section: Health-related Quality Of Lifementioning

confidence: 99%

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The Jamaican Haemophilia Registry: Describing the burden of disease

et al. 2018

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“…The Canadian Hemophilia Outcomes–Kids Life Assessment Tool (CHO‐KLAT) is a disease‐specific HRQoL instrument validated for use in boys 4‐18 years of age. It has been used in a number of clinical studies and was found to be sensitive to clinically important changes in the setting of the use of factor (F)VIII/FIX concentrates for the management of boys with hemophilia . Pooled CHO‐KLAT data from clinical studies in 9 countries provided a sample size large enough to allow evaluation of the impact of treatment on HRQoL while controlling for confounding relationships.…”

Section: Introductionmentioning

confidence: 99%

Impact of prophylaxis on health‐related quality of life of boys with hemophilia: An analysis of pooled data from 9 countries

Usuba

Price

Blanchette

et al. 2019

Research and Practice in Thrombosis and Haemostasis

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Background Prophylaxis reduces the frequency of bleeds in boys with severe hemophilia and is the standard care for their management in resource‐abundant countries. The effect of prophylaxis on Health‐Related Quality of Life (HRQoL) has not been established, because the sample sizes of most studies are too small to explore the relationship of multiple factors that influence HRQoL. Methods The aim of this study was to assess the impact of hemophilia severity and treatment regimen on HRQoL and to establish the minimum important difference (MID) using the international level of score distributions. HRQoL data were pooled from 7 studies across 9 countries. HRQoL was measured using the Canadian Hemophilia Outcomes–Kids’ Life Assessment Tool (CHO‐KLAT). A mixed‐effect linear regression analysis was employed to assess the impact of prophylaxis on the CHO‐KLAT score. Results Data from 401 boys with hemophilia were analyzed (57.6% severe hemophilia and 57.6% receiving prophylaxis). The model revealed that receiving prophylaxis was significantly associated with higher HRQoL (regression coefficient 8.5, 95% confidence interval [CI] 3.9‐13.1). Boys with severe hemophilia had a significantly lower HRQoL as compared to boys with moderate and mild hemophilia whose CHO‐KLAT scores were 7.0 and 6.6 points higher, respectively. There was a significant interaction between treatment and disease severity (P = 0.023), indicating prophylaxis has the most significant impact in boys with severe hemophilia. Based on these pooled data, the MID of the CHO‐KLAT was established at 6.5. Conclusions This study confirms the positive effect of prophylaxis on HRQoL in boys with hemophilia in a real‐world setting and provides initial benchmarks for interpreting HRQoL scores based on use of the CHO‐KLAT instrument.

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Pharmacokinetic variability of factor VIII concentrates in Chinese pediatric patients with moderate or severe hemophilia A

Chen

Huang

et al. 2021

Pediatric Investigation

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Importance The use of factor VIII (FVIII) concentrates under pharmacokinetic (PK) guidance has become the main approach for treatment of hemophilia. However, limited PK research has been conducted in Chinese pediatric patients. Objective To investigate the PK parameters of various FVIII concentrates in Chinese pediatric patients. Methods Seventy‐nine patients were enrolled (28 treated with Kogenate FS ® , 23 treated with Advate ® , and 28 treated with GreenMono™). All enrolled patients participated in single‐dose PK analysis after at least a 3‐day washout period. Blood samples were collected predose, as well as at 1 h, 9 h, 24 h, and 48 h after infusion; FVIII levels were measured using a one‐stage clotting assay. von Willebrand Factor Antigen (VWF:Ag) levels and blood types were also determined. PK parameters were evaluated by WAPPS‐Hemo. Results Mean values of terminal elimination half‐life time (t 1/2 ) for the Kogenate FS ® , Advate ® , and GreenMono™ FVIII groups were 12.24 h, 10.18 h, and 9.62 h; median clearance values were 4.16, 6.23, and 5.11 mL·kg −1 ·h −1 ; and median in vivo recovery values were 1.97, 1.55, and 1.61 IU/dL per IU/kg. Longer t 1/2 , higher in vivo recovery, and lower clearance were observed in patients with higher VWF:Ag level who were treated with recombinant concentrates. Interpretation Chinese pediatric patients with hemophilia had FVIII PK characteristics similar to those previously observed in non‐Chinese children, including large variation among individuals. VWF:Ag level and FVIII brand were associated with differences in FVIII PK. Thus, PK‐guided dosing should be used to optimize individualized therapy in Chinese children.

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A prospective study of health‐related quality of life of boys with severe haemophilia A in China: comparing on‐demand to prophylaxis treatment

Cited by 19 publications

References 25 publications

The Jamaican Haemophilia Registry: Describing the burden of disease

The Jamaican Haemophilia Registry: Describing the burden of disease

Impact of prophylaxis on health‐related quality of life of boys with hemophilia: An analysis of pooled data from 9 countries

Pharmacokinetic variability of factor VIII concentrates in Chinese pediatric patients with moderate or severe hemophilia A

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