Caregiver-Reported Economic Impacts of Pediatric Rare Diseases-A Scoping Review.

: Rare diseases are conditions that are individually rare but collectively common. These diseases can incur significant direct and indirect costs with a combination of high medical expenses, loss of income, and additional non-medical costs. Despite this, research into the economic cost for families of children with a rare disease is lacking.

Fathers' Ongoing Journey When a Child in the Family Has a Chronic Condition: A Meta-Synthesis.

This qualitative meta-synthesis was conducted with the aim to understand fathers' experiences and involvement when their child has a chronic condition within family context. Family nurse researchers from five countries identified 19 studies through a systematic search. Inclusion criteria were: (a) fathers as primary informant; (b) children (<19 years) with a chronic condition; (c) written in English, Spanish, French, or Portuguese.

Interventions to support young adults and families with the healthcare transition between paediatric and adult nephrology health services: A systematic scoping review.

Background: Healthcare transition can be challenging for young people and families living with chronic kidney conditions, including those with rare renal disorders who often have multi-systemic conditions, those who have undergone kidney transplantation, and those who face intense treatments like dialysis. Comprehensive, holistic healthcare transition interventions are required, encompassing physical, psychosocial, sexual, educational and vocational support.

Aim: This manuscript presents a systematic scoping review synthesising the healthcare transition interventions to support youth and families within nephrology services.

A qualitative exploration of children's lives with rare diseases.

Background: Rare diseases encompass a diverse group of debilitating and sometimes life-threatening conditions that affect a small percentage of the population, posing a significant public health challenge. Despite their rarity, around 70% of these diseases afflict children, yet limited research has focused on their experiences. This study aimed to gain insights into the day-to-day challenges children living with rare diseases face.

The economic impact of living with a rare disease for children and their families: a scoping review protocol.

Background: Rare diseases are an often chronic, progressive and life-limiting group of conditions affecting more than 30 million people in Europe. These diseases are associated with significant direct and indirect costs to a spectrum of stakeholders, ranging from individuals and their families to society overall. Further quantitative research on the economic cost for children and their families living with a rare disease is required as there is little known on this topic.