The cost of childhood cancer from the family's perspective: a critical review

The costs incurred by the families of children with cancer remain under researched. The objectives were to systematically review the literature and identify research and clinical implications. Thirteen studies were critically appraised using the Pediatric Quality Appraisal Questionnaire (PQAQ) [Ungar and Santos. Value Health 2003; 6:584-594]. Existing research indicates that families incur significant variable costs throughout cancer treatment. However, problems with the published studies related to various PQAQ domains (e.g., time horizon, sensitivity analysis) rendered the magnitude of families' childhood cancer costs somewhat uncertain. Strategies for enhancing the quality of childhood cancer cost of illness research are presented. Implications for clinical practice are discussed.     

A roadmap to surgery in osteogenesis imperfecta: results of an international collaboration of patient organizations and interdisciplinary care teams

Background and purpose — Involvement of patient organizations is steadily increasing in guidelines for treatment of various diseases and conditions for better care from the patient’s viewpoint and better comparability of outcomes. For this reason, the Osteogenesis Imperfecta Federation Europe and the Care4BrittleBones Foundation convened an interdisciplinary task force of 3 members from patient organizations and 12 healthcare professionals from recognized centers for interdisciplinary care for children and adults with osteogenesis imperfecta (OI) to develop guidelines for a basic roadmap to surgery in OI.Methods — All information from 9 telephone conferences, expert consultations, and face-to-face meetings during the International Conference for Quality of Life for Osteogenesis Imperfecta 2019 was used by the task force to define themes and associated recommendations.Results — Consensus on recommendations was reached within 4 themes: the interdisciplinary approach, the surgical decision-making conversation, surgical technique guidelines for OI, and the feedback loop after surgery.Interpretation — The basic guidelines of this roadmap for the interdisciplinary approach to surgical care in children and adults with OI is expected to improve standardization of clinical practice and comparability of outcomes across treatment centers.

Expert consensus remains the best available method for guiding surgical care in most rare diseases, due to the relative lack of evidence-based practices. With a prevalence between 1:10,000 and 1:20,000, osteogenesis imperfecta (OI) is a rare genetic disease affecting the quality and quantity of collagen I. Not only bone with frequent fractures and deformities, but all tissues containing collagen I are affected (Marini et al. 2017, Chougui et al. 2020). The somewhat unpredictable phenotypic variability of the disease is often grouped according to the clinical Sillence classification I–V (Van Dijk and Sillence 2014). However, each patient is unique not only in impairments but also in treatment needs. The most severe type III has the weakest bone and not all these individuals reach the level of standing and walking. Many patients undergo surgery more than once. On the initiative of the Osteogenesis Imperfecta Federation Europe (OIFE) and the Care4BrittleBones (Care4BB) Foundation, an international interdisciplinary task force was invited to create a roadmap for a standardized, integrated approach for optimal outcomes of surgery, not only from a surgical view, but also from the patient’s perspective.     

Tele-practice guidelines for the symptom management of children undergoing cancer treatment

The provision of tele-practice symptom management is often without the provision of evidence-based guidelines. Under the auspices of the Pediatric Oncology Group of Ontario, a nursing task force was established to appraise the evidence and develop guidelines. Promising new efforts to enhance symptom management through tele-practice are emerging. Seven guidelines and one documentation tool were created from evidence compiled from case reports, clinical examples, and nonexperimental studies. The symptom management guidelines contribute to the paucity of literature and may serve as a useful resource for health professionals providing telephone advice and conducting tele-practice symptom management assessments.     

The psychosocial experience of individuals living with osteogenesis imperfecta: a mixed-methods systematic review

Purpose

Osteogenesis imperfecta (OI) is a genetic disorder (prevalence: 1:10,000), leading to bone fragility, frequent fractures, and varying degrees of physical limitations. Despite a substantial amount of research on the genetics, pathophysiology, and treatments related to OI, there remains a paucity of knowledge concerning the lived psychosocial experience of the OI population. This mixed-methods systematic review aimed to review, appraise, and synthesize the literature on the psychosocial experience of children and adults with OI with the goal of identifying implications for research, practice, and policy-making.

Methods

Using a systematic methodology, quantitative, qualitative, and mixed-methods studies were accessed through database searching, screened, assessed for eligibility, and appraised. Data from the selected studies fulfilling the eligibility and quality criteria were extracted and synthesized using thematic analysis with an inductive approach.

Results

A total of four qualitative and 20 quantitative studies, with various study designs and methodologies ranging in quality, were included in the review (n = 800; comprising 610 children and 175 adults with OI types I, III, IV, and V, ten parents and five healthcare professionals). Six themes were identified: intellectual feats, isolation and feeling different, fear of fractures, coping with challenges, adapting by learning new skills, and social relationships.

Conclusion

These findings highlighted key aspects of the experiences of children and adults with OI and will be essential for improving the quality and direction of research, tailoring clinical interventions addressing the psychosocial needs and quality of life of individuals with OI, and raising awareness among caregivers, healthcare professionals, administrators, and policy-makers associated with the OI population.