From the voices of people with haemophilia A and their caregivers: Challenges with current treatment, their impact on quality of life and desired improvements in future therapies.

Introduction: Haemophilia A is a chronic disease requiring frequent intravenous infusions of recombinant factor VIII. Previous studies have shown that challenges associated with current treatments may have significant impacts on quality of life (QoL) that are as important as the health outcomes conferred by the therapy. Emerging therapeutic innovations offer the potential to mitigate treatment-related challenges, and it is therefore important to develop a better understanding of patient and caregiver experiences with existing haemophilia A treatments in order to characterize the full value of new treatments.

Symptom assessment in children receiving cancer therapy: the parents' perspective.

Goals Of Work: We aimed to develop an instrument to assess cancer-treatment-related adverse effects that parents believe children find most bothersome and use it to solicit the opinions of parents regarding this issue.

Materials And Methods: Parents of children 4 to 18 years of age who had received intravenous antineoplastic therapy in the last month were asked to rank prevalence, severity, and degree of bother of each symptom on behalf of their child using a questionnaire.

Main Results: One hundred fifty-eight of 200 (82%) questionnaires were evaluable.