Better Efforts to Evaluate Quality of Life in CTEPH Patients Needed, Review Finds

Magdalena Kegel avatar

by Magdalena Kegel |

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Quality of Life assessments

Quality of life is an important measure of patient health and treatment effectiveness, but often overlooked in people with chronic thromboembolic pulmonary hypertension (CTEPH). A review of such measurements in this patient group found plenty of room for improvement both in tools selected and their use — and called for more research on the topic.

On the rare occasions when quality of life measures are assessed in clinical practice and trials, the study revealed, those evaluations lack consistency in approach and methods. In the review, published in the European Respiratory Journal, researchers at Johns Hopkins University School of Medicine, Baltimore, took a closer look at previously published attempts to estimate quality of life in people with thromboembolic pulmonary hypertension, and the tools used for evaluation.

Under most circumstances, patients with pulmonary hypertension because of chronic lung embolisms undergo surgery to cure the condition. In 20 to 40 percent of these patients, however, surgery is not an option. And even among those who do, an estimated 17 to 35 percent continue having symptoms despite surgery.

People with CTEPH have traditionally been treated off-label with medications intended for pulmonary arterial hypertension. In 2013, the U.S. Food and Drug Administration (FDA) approved riociguat (Adempas) as the first drug specifically targeting thromboembolic PH patients. Given that recent date, there is a scarcity of clinical trials focusing on this patient group.

The study, Quality of life in patients with chronic thromboembolic pulmonary hypertension, found that general tools were more often used to assess quality of life than disease-specific ones, and while this can ease the comparisons between different disease types, the small number of patients in clinical trials restricted their feasibility.

Among the tools used with CTEPH patients, the PH-specific CAMPHOR is the only one validated in patients with thromboembolic PH, making comparisons between trials using different tools challenging.

Several retrospective studies comparing surgery and drug treatment have shown that patients have an improved quality of life after surgery. This improvement is most often accompanied by reduced symptoms and improved function, but improvement has also been reported by patients experiencing residual symptoms after surgery. This is in contrast to patients receiving medical treatment, which has shown a relatively modest impact on quality of life in the instances where it was evaluated.

The Phase 3 clinical trial on riociguat — the only study specifically investigating quality of life in thromboembolic PH patients — echoed findings from trials with mixed patient groups. It demonstrated a poor effect on quality of life, indicating the need for better drug treatments, as well as more robust ways to assess their efficacy.

Researchers concluded that further studies are required to assess quality of life in patients with CTEPH and to validate the optimal tools to determine this parameter.

A Conversation With Rare Disease Advocates