Researchers have developed a tool to assess quality of life (QoL) among patients with hereditary hemorrhagic telangiectasia (HHT). The hope is that it will help clinicians manage these patients and serve as a useful metric in clinical trials of HHT treatments, according to a report published in Blood Advances.
The simple, four-question survey was sent to patients registered with Cure HHT to elicit QoL measures, particularly the effects of nosebleeds on QoL since they are often the most disruptive symptom.
“In addition to the [QoL] score, there is also value in the score for the individual items, as they offer insight into what aspect of the disease is impacting an individual’s [QoL] most, which we will not get from a single question on severity,” said lead author Raj Kasthuri, MBBS, MD, of the University of North Carolina School of Medicine.
Given that changes to a patient’s clinical picture can affect the quality of a person’s life, having a reliable measure of QoL is a key tool for the best patient management, the researchers wrote. Existing measures of QoL for patients with HHT aren’t quite adequate, they added. For example, the Short Form-36, which is the most frequently used tool, is well-validated but does not specifically address HHT.
Disease-specific tools can “help determine the contribution of specific symptoms to limitations in physical functioning and may be more responsive to disease-specific therapies and interventions, making them more effective patient-reported outcomes for clinical trials and effectiveness research,” the investigators noted.
In the test survey, 45% of patients said that HHT had interrupted their job “quite a bit” or more, and 29% said the disorder “usually” or “always” interfered with family activities. Nearly one-quarter of those surveyed (24%) said they avoided social activities “usually” or “always,” and 31% said they “frequently” missed work or school or didn’t work at all because of HHT. Most survey respondents identified their disease as moderate (46%) to severe (25%).
Reliability of the tool was established, with a Cronbach’s alpha of 0.83. A principal components analysis found that the questions were “unidimensional,” said lead statistician Sonia Thomas, DrPH, of RTI International and the University of North Carolina. Unidimensionality means the questions measured the same latent variable so that, taken together, they analyze the desired insights about the patient’s QoL.
Use of the tool in an ongoing clinical trial funded by the National Institutes of Health evaluating the effect of pomalidomide to control bleeding in HHT will provide further refinement and validation of the tool. According to Dr. Kasthuri, if the medication is effective, it will tell investigators whether improving bleeding related to HHT helps improve QoL.
At his center, providers have started to use the tool to monitor patients’ QoL. “The simplicity and ease of administration makes [this instrument] easy to incorporate as part of routine clinic visits and may provide valuable information,” he said.
Any conflicts of interest declared by the authors can be found in the original article.
Reference
Kasthuri R, Chaturvedi S, Thomas S, et al. Development and performance of a hereditary hemorrhagic telangiectasia specific quality of life instrument [published online July 22]. Blood Adv. doi: 10.1182/bloodadvances.2022007748.