Quality of Life, Self-reported Outcomes and Impact of Education Among People With Moderate and Severe Hemophilia A: an Integrated Perspective From a Latin American Country
Liliana Torres, Oscar Peñuela, Maria del Rosario Forero, Juan Satizabal, Ximena Salazar,Diana Benavides, Raul Gamarra, Marcela Rivera, David Vizcaya, Juan-Sebastian Franco
Collecting and interpreting self-reported outcomes among people with hemophilia A supports the understanding of the burden of the disease and its treatment to improve holistic care. However, in Colombia, this information is limited. Therefore, this study aimed to describe the knowledge, perception and burden of hemophilia A from the patients’ perspective. A cross-sectional study was conducted in the context of a hemophilia educational bootcamp held from November 29th to December 1st, 2019, in Medellin, Colombia. The bootcamp was organized by a hemophilia patient association responsible for contacting and inviting patients with hemophilia A (PwHA). Information on patients’ health beliefs, treatment experiences, and health-related quality of life (HRQoL) was obtained through focus groups, individual interviews and the Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire. A total of 25 moderate or severe PwHA were enrolled in this study and completed the PROBE questionnaire.
Hemophilia A is a rare hereditary bleeding disorder caused by a deficiency of coagulation factor VIII (FVIII) and characterized by repeated and prolonged bleeding into muscles and joints that lead to pain, physical limitations and a negative impact on health-related quality of life (HRQoL) . Prophylaxis is the preferred treatment for patients with severe hemophilia A (PwHA), given its positive impact on joint bleeding events, arthropathy, absenteeism, physical health, pain and HRQoL [2, 3]. However, a considerable number of PwHA have suboptimal adherence to treatment, increasing the probability of bleeding events and long-term disability [1, 4]. Several barriers to adherence are related to the patient condition, assigned treatment, health care system, and socioeconomic aspects, especially in countries that suffer from large social inequalities, such as Latin American countries . In addition, health beliefs around the disease and treatment have been associated with the patient’s experience, influencing their understanding of the diagnosis, the prognosis, and the potential benefits of prophylaxis treatment [1, 6], reinforcing the need for personalized treatment strategies for prophylaxis therapy . In this regard, disease-specific bootcamps act as educational initiatives for PwHA to allow them to speak openly, ask questions, confide and learn factual information about this disorder [8, 9].
Materials and method
The bootcamp included 30 PwHA whose ages ranged from 10 to 59 years. Patients aged <18 were accompanied by one of their parents or legal guardians. This 3-day camp was designed to encourage participants to meet other PwHA, share experiences and increase knowledge of the disease. The bootcamp was coordinated by a hemophilia patient organization (Liga Antioqueña de Hemofilia) and sponsored by Bayer. The hemophilia patient organization was responsible for contacting and inviting PwHA to the bootcamp, following local regulations. During the first day, to conduct bonding activities, the participants were randomly distributed into three different groups. On the second day, educational activities were performed, as well as recreational activities. On the third and last day, qualitative interviews and closure of the camp took place. The activities were guided by three counsellors: two social workers and a nurse. Additionally, the campsite had two paramedics and an ambulance available at any time. The design and conduct of the present study were an add-on to the bootcamp given the unique opportunity to gather evidence in a patient-centric environment.
Twenty-four participants had severe hemophilia A (96%), and the remaining patients had moderate hemophilia A. The median age was 20 years (IQR, 13–30 years), and most participants came from urban areas (60%; n = 15), with the main cities being Medellin, Cali, Bucaramanga and Villavicencio; the remaining participants (40%; n = 10) were from suburban/rural areas in the Antioquia and Santander regions. The median weight was 62 kg (IQR: 51–72 kg), and the median number of years of schooling was 10 (IQR: 6.75–11.25 years) (Table 1). Five individuals participated in the bootcamp but were not eligible for this observational study: two with hemophilia B, two with mild hemophilia A and one with von Willebrand disease.
Our study thoroughly described a cohort of PwHA from Colombia, including demographic and clinical characteristics as well as patient-reported outcomes and patient beliefs and experiences related to their understanding of the disease and its treatment. Overall, patients reported an acceptable HRQoL, with worse scores observed in the domains of mobility and pain/discomfort. These results were consistent with those from our qualitative data analysis. PwHA reported experiencing challenges regarding their capacity to act independently daily and their access to care and medicines. Some patients learned to self-infuse prophylaxis treatment because of challenges in reaching a trained health professional who could do so in their living area. As an alternative to the health care system, patients accessed non-health care institutions such as patient associations to provide them with legal advice and psychosocial support.
The value of involving PwHA and their perspectives in the decision-making process of their disease and treatment has gained increasing relevance in recent years. This study explored the perspectives of PwHA regarding their disease and treatment experience in the context of a disease-specific educational bootcamp and provided unique insights into their disease knowledge, QoL and expected treatment outcomes. The outcomes of this study could serve as key complementary information to health systems and policy-makers to implement a patient-centered framework for public health decision-making that includes a comprehensive analysis of the value and impact of novel hemophilia therapies such as EHL FVIII products, which have the potential to decrease the treatment burden and improve adherence, resulting in better health outcomes.
The study team wish to extend a special thanks to the “Liga Antioqueña de Hemofilia” for their support in coordinating and conducting the Hemophilia Patient Bootcamp, and to the study participants who provided valuable insight into their experience with the disease and its treatment. The authors would also like to acknowledge the support of Dr. Mark Skinner and the PROBE study for the review of the manuscript.
Citation: Torres L, Peñuela O, Forero MdR, Satizabal J, Salazar X, Benavides D, et al. (2023) Quality of life, self-reported outcomes and impact of education among people with moderate and severe hemophilia A: An integrated perspective from a Latin American country. PLoS ONE 18(7): e0287972. https://doi.org/10.1371/journal.pone.0287972
Editor: Wolfgang Miesbach, University Hospital Frankfurt, GERMANY
Received: September 5, 2022; Accepted: June 16, 2023; Published: July 6, 2023
Copyright: © 2023 Torres et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.
Data Availability: All relevant data are within the manuscript and its Supporting Information files.
Funding: The study was funded by Bayer SA Colombia. Medical writing was provided by Ana Maria Perez and editorial assistance was provided by American Journal Experts, North Carolina, USA, funded by Bayer SA Colombia. This study was conducted in accordance with Good Pharmacoepidemiology Practices. The study protocol was approved by an expert committee from Bayer prior to study initiation and the analysis dataset is available as supplementary material.
Competing interests: Juan Satizabal, Ximena Salazar, Diana Benavides and Raul Gamarra are employees of IPSOS Napoleon Franco S.A. Liliana Torres, Oscar Peñuela, Maria del Rosario Forero, Marcela Rivera, David Vizcaya and Juan-Sebastian Franco are employees of Bayer. All authors have no further conflicts to disclose. This does not alter our adherence to PLOS ONE policies on sharing data and materials.