Quality of Life, Clinical Effectiveness, and Satisfaction in Pediatric and Young Adult Patients with Sickle Cell Disease Receiving Hydroxyurea Therapy

Abstract

A AB BS ST TR RA AC CT T O Ob bj je ec ct ti iv ve e: : Despite documented benefits of hydroxyurea (HU) for pediatric and young adults with sickle cell disease (SCD), there is still limited experience with the use of HU in these age groups and knowledge regarding its long-term efficacy, safety, and acceptance remains poorly defined. This study aims to evaluate quality of life, clinical effectiveness, and satisfaction in pediatric and young adult patients with SCD receiving HU. M Ma at te er ri ia al l a an nd d M Me et th ho od ds s: : In this study, 34 pediatric (7-11 and 12-17 years) and 16 young adult (18-22 years) patients with SCD receiving HU for at least a year were participated. The data for effectiveness of hydroxyurea therapy and parameters that may affect compliance to treatment and life quality of the participants were evaluated with using Demographic Data Collection Form, Life Quality Survey Short Form-36 (SF-36), Child Health Questionnaire-Parent Form 50, Case Report Form and HU Satisfaction Survey. R Re es su ul lt ts s: : The patients aged 7-11, 12-17, and 18-22 years were being treated with HU at 15.3±4.0 (for 4.7±2.7 years), 14.4±3.7 (for 5.7±3.3 years), and 14.0±6.4 mg/kg/day (for 5.1±2.6 years), respectively. Correlation studies revealed that HU dose was not significantly correlated SF-36 summary scores in these patients. Duration of HU therapy was positively correlated with the SF-36 summary scores for physical functioning, physical role limitation, mental health, and general health perception in the young adult patients. However, duration of HU therapy was negatively correlated with the perceived effectiveness of therapy while positive correlations were observed between duration of treatment and burden of therapy/side effects. C Co on nc cl lu us si io on n: : These findings suggest that the health quality and compliance of the pediatric and young adult patients to therapy might be low due to not sufficiently effective HU therapy in addition to comorbidities, concomitant drug use, and side effects. K Ke ey yw wo or rd ds s: : Hydroxyurea; anemia, sickle cell; compliance Ö ÖZ ZE ET T A Am ma aç ç: : Orak hücre hastalığı (OHH) olan pediatrik ve genç erişkin hastalarda hidroksiüre (HÜ)'nün yararlarının biliniyor olmasına karşın, bu yaş gruplarında HÜ'nün kullanımıyla ilgili deneyim hala sınırlıdır ve uzun süreli etkililiği, güvenliği ve kabul edilebilirliğine ilişkin bilgiler yetersiz kalmıştır. Bu çalışmada OHH nedeniyle HÜ tedavisi gören pediatrik ve genç erişkin hastalarda yaşam kalitesi, klinik etkinlik ve tedaviye uyum değerlendirilmiştir. G Ge er re eç ç v ve e Y Yö ön nt te em ml le er r: : Bu çalış-maya, OHH nedeniyle en az 1 yıl süre HÜ tedavisi gören 34 pediatrik (7-11 ve 12-17 yaş) ile 16 genç erişkin (18-22 yaş) hasta katılmıştır. HÜ tedavisinin etkinliği ve katılımcıların tedaviye uyuncu ile yaşam kalitesine ilişkin veriler Demografik Veri Toplama Formu, Yaşam Kalitesi Anketi Kısa Form-36 (KF-36), Çocuk Sağlığı Anketi (Anne/Baba Raporu) 50, Olgu Rapor Formu ve HÜ Tedavisinden Memn...

Health-Related Quality of Life of Children and Adolescents with Sickle Cell Disease in the Middle East and North Africa Region

“… 43 According to Cevher et al ., both quality of life and adherence to HU therapy was low among paediatric and young adult patients in Turkey; this was attributed to the lack of effectiveness of HU therapy, in addition to comorbidity, concomitant drug use and side-effects. 47 …”

“…A total of 10 articles were included in the systematic review [Table 1]. 21,[43][44][45][46][47][48][49][50][51] Of these, nine were observational studies, including five cross-sectional, two case-control and two cohort studies. 21,[44][45][46][47][48][49][50][51] The *Methodological quality was assessed using an adapted version of the Newcastle-Ottawa Quality Assessment Scale, except for mixed-method studies, which were assessed using the Mixed Methods Appraisal Tool.…”

“…21,[43][44][45][46][47][48][49][50][51] Of these, nine were observational studies, including five cross-sectional, two case-control and two cohort studies. 21,[44][45][46][47][48][49][50][51] The *Methodological quality was assessed using an adapted version of the Newcastle-Ottawa Quality Assessment Scale, except for mixed-method studies, which were assessed using the Mixed Methods Appraisal Tool. 41,42 Cross-sectional, cohort and case-control studies were scored out of 8, 6 and 9, respectively, with scores of <50% considered to indicate poor methodological quality.…”

“…Four studies were conducted in Saudi Arabia, two in Turkey and one each in Iraq, Sudan, Uganda and Oman. 21,[43][44][45][46][47][48][49][50][51] Most of the studies focused primarily on children and adolescents aged 2-18 years. 21,[43][44][45][46]48,50,51 However, two studies focused both on paediatric and young adult populations aged 18-22 years.…”

“…21,[43][44][45][46]48,50,51 However, two studies focused both on paediatric and young adult populations aged 18-22 years. 47,49 In most cases, self-reported measures were applied to children over the age of 8 years who were literate and capable of comprehension, with caregiver proxy reports utilised for those between 2-8 years. 21,[43][44][45][46][47][49][50][51] The sample size for children and adolescents with SCD or SCA ranged from 37-180.…”