Longitudinal Changes in Health-Related Quality of Life for Chronic Diseases: An Example in Hemophilia A

Poon, Jiat Ling; Doctor, Jason N.; Nichol, Michael B.

doi:10.1007/s11606-014-2893-y

Cited by 24 publications

(41 citation statements)

References 17 publications

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“…In our patients with haemophilia, the presence of target joints led to a low HRQOL related to both psychological and physical states. This finding was consistent with previous reports on negative factors affecting the social life of children . Therefore, it seemed possible that a greater awareness of target joints during routine comprehensive haemophilia care could improve QOL in these individuals.…”

Section: Discussionsupporting

confidence: 92%

“…In particular, the development of an inhibitor alloantibody against factor VIII or IX and articular deformities markedly affected HRQOL. Unexpected bleeding, frequent hemarthroses, school absences and unplanned hospital visits were also reported as significant risk factors for low HRQOL . In circumstances in which haemophilia treatment now aims to improve the ‘normal activity of life’, comparing the age‐matched haemophiliacs with the non‐haemophiliacs is important.…”

Section: Introductionmentioning

confidence: 99%

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Assessment of self‐/parent‐reported quality of life in Japanese children with haemophilia using the Japanese version of KIDSCREEN‐52

et al. 2020

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Introduction Assessing health‐related quality of life (HRQOL) is critical for providing comprehensive clinical care to patients with haemophilia. HRQOL in individuals with similar cultural backgrounds should be compared using internationally standardized, generic questionnaires. Aim To evaluate self‐/parent‐assessed HRQOL in Japanese children and adolescents with haemophilia A or B. Methods Children and adolescents aged 8‐18 years were enrolled. The haemophilia group comprised families with haemophilia, and the control group comprised those without chronic illness. HRQOL was assessed using the self‐/parent‐reported questionnaire KIDSCREEN‐52, the Japanese version. The Oslo 3‐Item Social Support Scale was investigated. Results The questionnaire was completed by 36 families in the haemophilia group and 160 parents and children in the control group. Haemophilia children aged 8‐12 years had lower scores for ‘moods and emotions’ than control children; the parents had lower scores in the haemophilia group than in the control group for ‘moods and emotions’, ‘social support and peers’, and ‘school environment’. No significant differences in HRQOL were observed between both groups of adolescents aged 13‐18 years or their parents. Neck‐shoulder pain was associated with a low psychological state, including ‘self‐perception’, but other joint pains did not affect the outcomes of the HRQOL indices. Social support weaknesses were associated with low physical and psychological states, whereas unexpected hospital visits identified low values for ‘self‐perception’, ‘autonomy’, and ‘school environment’. Conclusion Proactive mental and clinical care in haemophilia families, especially with young children, will foster a better environment for patients and their parents and ease concerns about progress in haemophilia.

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Section: Discussionsupporting

confidence: 92%

Section: Introductionmentioning

confidence: 99%

Assessment of self‐/parent‐reported quality of life in Japanese children with haemophilia using the Japanese version of KIDSCREEN‐52

et al. 2020

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“…Haemophilia A, characterized by a deficiency of coagulation factor VIII (FVIII) activity, leads to a lifelong tendency of bleeding, related arthropathy and diminished quality of life (QoL) . Intravenous FVIII replacement given prophylactically is the current standard of care for persons with haemophilia A (PwHA).…”

Section: Introductionmentioning

confidence: 99%

“…Haemophilia A, characterized by a deficiency of coagulation factor VIII (FVIII) activity, leads to a lifelong tendency of bleeding, 1 related arthropathy and diminished quality of life (QoL). 2,3 Intravenous FVIII replacement given prophylactically is the current standard of care for persons with haemophilia A (PwHA). However, due to short FVIII half-life, regular intravenous infusions (2-4 times/wk) are required to maintain protective trough levels and achieve adequate haemostatic coverage.…”

Section: Introductionmentioning

confidence: 99%

Bleeding and safety outcomes in persons with haemophilia A without inhibitors: Results from a prospective non‐interventional study in a real‐world setting

et al. 2019

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Introduction Prospectively collected real‐world data on bleeds, haemophilia treatment and safety in persons with haemophilia A (PwHA) without factor VIII (FVIII) inhibitors are limited. A global, non‐interventional study (NIS; NCT02476942) prospectively collected real‐world data in PwHA who were treated per local routine clinical practice. Aim Assess annualized bleeding rate (ABR), haemophilia treatment practices and adverse events (AEs) in adult/adolescent PwHA without inhibitors. Methods Eligible participants aged ≥12 years with severe HA without history of inhibitors prospectively collected bleeding and treatment information. Results Ninety‐four participants were enrolled (median [range] age, 34 [12‐76] years) and monitored for a median (range) of 29.8 (12.4‐47.7) weeks. In the episodic (n = 45) and prophylactic (n = 49) treatment groups, respectively, 872/1066 (81.8%) and 151/189 (79.9%), bleeds were treated; ABRs (95% confidence interval) were 36.1 (30.8‐42.3) and 5.0 (3.3‐7.5), respectively, for treated bleeds and 43.1 (36.5‐50.9) and 6.2 (4.2‐9.2), respectively, for all bleeds, and median (interquartile range) ABRs were 31.1 (19.8‐51.6) and 1.9 (0.0‐8.2), respectively, for treated bleeds and 35.3 (21.7‐62.9) and 2.7 (0.0‐9.4), respectively, for all bleeds. Half of the participants on FVIII prophylaxis had relatively high adherence to treatment, using 2.9 and 2.1 median doses/wk of standard and extended half‐life FVIII, respectively. Serious AEs included gastrointestinal polyp haemorrhage and haemarthrosis; the most common AE was viral upper respiratory tract infection. Conclusion PwHA without inhibitors continue to bleed on prophylaxis, consistent with the literature, and require treatment for breakthrough bleeds. This prospective NIS demonstrates the need for more efficacious haemostatic approaches.

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“…Haemophilia A is characterized by a coagulation factor VIII (FVIII) deficiency, leaving individuals at high risk of frequent bleeding, especially in the joints, leading to arthropathy and poor quality of life . Replacement therapy with FVIII concentrate is the standard of care for persons with haemophilia A (PwHA).…”

Section: Introductionmentioning

confidence: 99%

Bleeding events and safety outcomes in persons with haemophilia A with inhibitors: A prospective, multi‐centre, non‐interventional study

Mahlangu

Oldenburg²,

Callaghan

et al. 2018

Haemophilia

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Introduction Prospectively collected, real‐world data on bleeds, haemophilic treatment and safety outcomes in persons with haemophilia A (PwHA) with factor VIII (FVIII) inhibitors are limited. A prospective, global, multi‐centre, non‐interventional study (NIS; NCT02476942) collected detailed real‐world data in PwHA treated per local routine clinical practice. Aim To characterize bleeding rates, haemophilic treatment practices, prophylaxis adherence and adverse events (AEs) in adult/adolescent PwHA with inhibitors in the NIS. Methods Participants aged ≥12 years with congenital haemophilia A/documented high‐titre FVIII inhibitor history were enrolled. Participants remained on their usual treatment; no interventions were applied. Results Overall, 103 PwHA with inhibitors enrolled, (median [range] age 31 [12‐75] years) and were monitored for median (range) 26.0 (4.1‐69.6) weeks. In the episodic (n = 75) and prophylactic (n = 28) treatment groups, respectively, 1244 and 325 bleeds were reported, and 528 (42.4%) and 104 (32.0%) were not treated; annualized bleeding rates (ABRs; 95% confidence interval) were 18.6 (15.2‐22.8) and 14.9 (10.5‐21.2) for treated bleeds, and 32.7 (27.3‐39.1) and 25.0 (18.4‐34.0) for all bleeds. Coagulation products used included activated prothrombin complex concentrate (aPCC) and/or recombinant activated FVII. Among participants prescribed aPCC prophylaxis, 35.0% adhered to both prescribed frequency of aPCC administration and prescribed dose. Serious AEs of haemarthrosis and muscle haemorrhage were reported; most common AEs were arthralgia, viral upper respiratory tract infection and pyrexia. Conclusions ABRs (treated bleeds and all bleeds) remain high on standard treatment; this prospective NIS demonstrates the need for more effective treatments for PwHA with inhibitors to reduce/prevent bleeds, with potential to improve prophylaxis adherence and further improve outcomes.

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Longitudinal Changes in Health-Related Quality of Life for Chronic Diseases: An Example in Hemophilia A

Cited by 24 publications

References 17 publications

Assessment of self‐/parent‐reported quality of life in Japanese children with haemophilia using the Japanese version of KIDSCREEN‐52

Assessment of self‐/parent‐reported quality of life in Japanese children with haemophilia using the Japanese version of KIDSCREEN‐52

Bleeding and safety outcomes in persons with haemophilia A without inhibitors: Results from a prospective non‐interventional study in a real‐world setting

Bleeding events and safety outcomes in persons with haemophilia A with inhibitors: A prospective, multi‐centre, non‐interventional study

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