The likelihood that teenagers and young adults with hemophilia will use a prophylactic replacement therapy as prescribed is mostly influenced by their level of responsibility in taking their infusions, and their own estimates of risk for each activity, a study suggests.
To improve treatment adherence, doctors should discuss its importance during bleeding assessment visits, rather than regular visits, when young people are most receptive to such conversations, the researchers said.
The study “The Perspectives of Adolescents and Young Adults on Adherence to Prophylaxis in Hemophilia: A Qualitative Study” was published in the journal Patient Preference and Adherence.
Adolescents and young adults with a chronic illness like a bleeding disorder face a challenge in accepting responsibility for their disease and its treatment. “Notably, self-management skills must be learned during adolescence,” the researchers wrote.
Patients with severe hemophilia — marked by very low levels of clotting factor VIII or IX — need preventive treatment (prophylaxis) that requires a high level of compliance with the prescribed dose and frequency of treatment.
Replacement therapy consists of injecting concentrates of the missing clotting factor into the vein (intravenous infusions) twice weekly to every other day depending on therapy type, to maintain minimal clotting factor levels and prevent bleeding.
The therapy is started when the first bleeding occurs, typically between ages 2 to 4, and continues for life. Parents and patients can be trained to administer treatment at home, which is especially important for those with severe disease who need rapid treatment.
But complying with such a continuous regimen can be particularly difficult for younger patients, beginning their independence but a life phase marked by physical and emotional changes and potential rebellion against advice from parents or health care providers.
In fact, adherence to prophylaxis drops during adolescence — between 13% and 17% non-adherent, the study noted — which can lead to more breakthrough bleedings, damage to joints, more days missed at school and work, and a lower quality of life.
To understand the reasons for non-adherence from the perspective of teenagers and young adults with hemophilia, researchers at University Medical Center Utrecht and the University of Applied Sciences, both in the Netherlands, interviewed a group of Dutch patients, ages 14 to 24, using prophylaxis.
A total of 21 interviews involving 18 patients, including focus group and individual interviews, addressed perceived barriers, motivators, facilitators, and individual decision-making processes regarding prophylaxis.
Eleven of these 18 people had been classified as adherent to preventive therapy, four were sub-optimal, and three were non-adherent.
Two main factors were seen to affect treatment adherence — the varying levels of responsibility taken by the patient and parental support, and patients’ estimates of bleeding risk while engaging in certain activities.
Three distinct phases in terms of assuming responsibility for treatment and defining risk were noted as patients age and mature. In general, these phases mark periods where support from parents decreases and patient’s responsibility increases, as does the risk of non-adherence.
In the first phase, patients felt able to infuse themselves but parents were in charge most of the time. Given the high level of parental support, most adhered well to prophylaxis and voiced experiencing minimal or no bleeding episodes.
During the second phase, patients prepared and took the treatment on their own. Sometimes they skipped or forgot injections, despite reminders from parents who, they felt, were “too careful.” Self-estimates of their need for infusions were not always in line with their parents’ opinions, but parental advice was sometimes sought.
In this second phase of growing personal responsibility and risk estimation, adherence varied depending on the individual, and some reported experiencing bleeds.
In the third phase, patients assumed total responsibility for their treatment and parental advice was no longer involved (except in emergencies). Adherence ranged from those who regularly used prophylactic treatment (or took it immediately, if forgotten) to those who intentionally skipped treatment because they felt no need for it. “AYAs explained that they were weighing arguments in favor of and against prophylaxis,” the study noted.
Again, varying levels of adherence were reported, and “nearly all” 18 patients in this phase had “experience with bleeding.”
Self-estimates of risk were largely determined, according to interview responses, by weighing a desire to live like “healthy peers” against the risk of uncontrolled bleeding with or without prophylaxis. Not surprisingly, those who experienced minimal bleeding or a minimal impact of bleeds on their daily activities were less likely to be adherent than were those with bleeds after activities, especially bleeding that “substantially” affected daily life.
But all mentioned choosing to skip activities they deemed unsafe, even with prophylaxis, indicating “that feeling safe was more important than doing what they preferred,” the researchers noted.
“As such, we believe that patient education combined with MI [a motivational interview] should be applied during a bleeding assessment visit rather than a regular visit since the AYA [adolescent or young adult] is more open to learning,” they concluded.
Future research should focus on individual patient education at each phase defined in this study, and on motivational interviews to improve adherence, the research team added.
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