Clinical and economic burden of immune tolerance induction in entire patients with hemophilia A: Insights from a real-world Korean setting.

INTRODUCTION: The most notable challenge facing hemophilia A treatment is the development of inhibitors against factor VIII, resulting in increased clinical and socioeconomic burdens due to the need for expensive bypassing agents (BPAs). Although immune tolerance induction (ITI) is currently the primary approach for inhibiting and reducing the inhibitors, the lengthy duration of ITI necessitates the continued use of BPA to manage bleeding episodes. In this study, we aimed to obtain real-world evidence on the clinical and economic aspects and associated burdens experienced by patients with hemophilia A with inhibitors undergoing ITI in Korea. METHODS: Claims data from January 1, 2007, to December 31, 2020, were used in this study. The study cohort comprised patients with hemophilia A undergoing ITI, who were categorized into three groups: successful, failed, or continuation of ITI. We evaluated clinical and economic burdens, including monthly healthcare visits, medication costs, and total medical expenses. RESULTS: The study involved 33 cases of ITI across 32 patients. Excluding seven continuation cases where success could not be determined at the observation point, the estimated success rate of ITI was 80.8 %. The median duration of ITI for all patients was 25.7 months. While no significant disparities were noted in the ITI duration between successful and unsuccessful cases (24.51 vs. 25.66 months), substantial discrepancies were observed in the duration of BPA usage (11.10 vs. 25.66 months) and the number of prescribed BPAs (1.79 vs. 2.97). CONCLUSION: Successful ITI reduced both clinical and economic burdens, resulting in decreased monthly medication expenses and overall medical costs.

Factors associated with the comprehensive needs of caregivers of childhood cancer survivors in Korea.

PURPOSE: Caregivers of childhood cancer survivors (CCS) have diverse needs, which should be addressed to provide comprehensive cancer care. We aimed to evaluate the unmet needs of caregivers of CCS. METHODS: The subjects were 700 caregivers recruited at three major hospitals in South Korea. We collected study data using self-administered questionnaires and a thorough review of medical records. We assessed the unmet needs of caregivers using the comprehensive needs assessment tool for cancer caregivers and evaluated factors associated with the highest tertile range of unmet needs by multiple logistic regression analysis. RESULTS: The greatest unmet needs of caregivers had to do with healthcare staff, followed by information. Compared with father-caregivers, mother-caregivers had greater unmet needs related to health and psychological problems, family/social support, and religious/spiritual support, with odds ratios (95% confidence interval) of 3.79 (2.52-5.69), 3.17 (2.09-4.81), and 1.69 (1.14-2.50), respectively. Compared with caregivers of the youngest CCS (< 6 years), caregivers of CCS aged 12-18 years and caregivers of the oldest CCS (≥ 19 years) respectively showed 2.62 (1.24-5.52) and 3.18 (1.34-7.55) times greater unmet needs for information. Caregivers of CCS who received haematopoietic stem-cell transplantation had a 2.01-fold (1.14-3.57) greater need for practical support. CONCLUSION: Caregivers of CCS had substantial unmet needs required for comprehensive care for CCS. Several individual characteristics of caregivers and their children were significantly associated with greater unmet needs of the caregivers. IMPLICATIONS FOR CANCER SURVIVORS: Personalized support based on the characteristics of both CCS and their caregivers is required to provide comprehensive care for CCS.