Health related quality of life in children with sickle cell disease: A systematic review and meta-analysis

This study aimed to synthesize the evidence on the effects of disease‐modifying agents for managing sickle cell disease (SCD) in children and adolescents by means of a systematic review with network meta‐analyses, surface under the cumulative ranking curve (SUCRA) and stochastic multicriteria acceptability analyses (SMAA) (CRD42022328471). Eightteen randomized controlled trials (hydroxyurea [n = 7], l‐arginine [n = 3], antiplatelets [n = 2], immunotherapy/monoclonal antibodies [n = 2], sulfates [n = 2], docosahexaenoic acid [n = 1], niprisan [n = 1]) were analyzed. SUCRA and SMAA demonstrated that hydroxyurea at higher doses (30 mg/kg/day) or at fixed doses (20 mg/kg/day) and immunotherapy/monoclonal antibodies are more effective for preventing vaso‐occlusive crisis (i.e., lower probabilities of incidence of this event; 14, 25, and 30%, respectively), acute chest syndrome (probabilities ranging from 8 to 30%), and needing of transfusions (11–31%), while l‐arginine (100–200 mg/kg) and placebo were more prone to these events. Therapies were overall considered safe; however, antiplatelets and sulfates may lead to more severe adverse events. Although the evidence was graded as insufficient and weak, hydroxyurea remains the standard of care for this population, especially if a maximum tolerated dose schedule is considered.

Section: Discussionmentioning

confidence: 99%

Efficacy and safety of pharmacological interventions for managing sickle cell disease complications in children and adolescents: Systematic review with network meta‐analysis

Tonin

Ginete

Ferreira

et al. 2023

“…One recent systematic review showed that quality of life was significantly lower in children diagnosed with SCD compared to peers. 34 However, another review demonstrated large positive quality-of-life improvements reported by adult recipients post HCT. 35 Similar improvements have been shown in children post HCT.…”

Section: Discussionmentioning

confidence: 99%

“…Quality of life as rated by children was higher, but less so as compared to their parents’ ratings. One recent systematic review showed that quality of life was significantly lower in children diagnosed with SCD compared to peers 34 . However, another review demonstrated large positive quality‐of‐life improvements reported by adult recipients post HCT 35 .…”

Section: Discussionmentioning

confidence: 99%

Neuropsychological, behavioral, and quality‐of‐life outcomes in children and adolescents with sickle cell disease treated with nonmyeloablative matched sibling donor hematopoietic cell transplantation: A case series

Fay‐McClymont

Monagel

Singh

et al. 2022

Self Cite

“…[22][23][24][25][26][27] Children with SCD had a substantially lower HRQoL compared to both the general pediatric population, and other children with chronic disorders. 24,26,[28][29][30] The painful VOC strongly affects the HRQoL more than any other SCD-related complication, such as avascular necrosis or stroke. 31 This is likely due to lack of control, effective therapeutic interventions, 28 and the difficulty of pain management.…”

Section: Health-related Quality Of Lifementioning

confidence: 99%

Impact of hospitalization for vaso‐occlusive crisis on health‐related quality of life in children with sickle cell disease

Vuong,

Moussa,

van Muilekom

et al. 2023

BackgroundSickle cell disease (SCD) is characterized by vaso‐occlusive crises (VOCs) that impair the health‐related quality of life (HRQoL). The aim of this study is to evaluate the impact of hospitalization for VOCs on HRQoL in children with SCD over time.MethodsIn this longitudinal cohort study, children aged 8–18 years diagnosed with SCD at the Amsterdam UMC were included between 2012 and 2021. HRQoL was annually measured as part of standard care using the Pediatric Quality of Life Inventory. The impact of hospitalization for VOC on HRQoL was evaluated using linear mixed models 3, 6, 9, and 12 months after hospitalization. The effect of frequency of hospitalization for VOC on HRQoL was evaluated over the last 12 months.ResultsIn total, 94 children with SCD were included with a median age of 11.8 years (interquartile range [IQR]: 9–14). Thirty‐seven patients (39%) had been hospitalized for a VOC. Hospitalization for VOC led to a decrease of 3.2–4.8 points in total HRQoL compared to patients without hospitalization, most pronounced 3 months after hospitalization. Recurrent admission for VOC in the last 12 months was associated with a decrease of 2.3 points in total HRQoL (p = .04). The most affected subscale was physical functioning.ConclusionThe adverse effects of hospitalization for VOC in children with SCD persist up to 12 months after hospitalization. After hospitalization for VOC, extra attention and support for its negative impact on HRQoL are recommended. This study also underlines the importance of systematically measuring HRQoL, allowing clinicians to intervene accordingly.