A Novel Health-Related Quality of Life Assessment Tool for Nontransfusion-Dependent Beta Thalassemia

A new tool for measuring patient-reported outcomes (PROs) has been developed for patients with nontransfusion-dependent beta thalassemia (NTDT) and was presented in the American Journal of Hematology.

Other health-related quality of life (HRQoL) measures may not sufficiently address issues unique to NTDT, according to the researchers who developed the tool. The tool, called the NTDT-PRO^©, focuses on ranking severity of weakness, tiredness, and shortness of breath, with or without physical activity for each, for patients with NTDT.

The researchers conducted a multicenter, observational study examining responses to questionnaires completed over at least 24 weeks for 48 patients with NTDT in Italy, Lebanon, Greece, and Thailand. The NTDT-PRO was completed daily using a handheld device and was compared with the Functional Assessment of Cancer Therapy-Anemia (FACT-An) and 36-Item Short Form Health Survey version 2 (SF-36v2), which were administered at baseline and every 3 weeks thereafter. For the latter questionnaires, higher values reflected higher levels of function; for the NTDT-PRO, higher values reflected worse symptoms. Responses were compared between questionnaires during weeks 1, 3, and 12.

Responses to the NTDT-PRO showed negative correlations of varying strength and significance with metrics on the FACT-An and SF-36v2 scales, indicating that symptom severity on the NTDT-PRO exhibited some strong associations with decreased functionality across questionnaires.

The NTDT-PRO demonstrated high internal consistency and test-retest reliability. Weakness and tiredness were strongly correlated (r_s = 0.96, with physical activity for each; r_s = 0.89, without physical activity for each). Weakness, tiredness, and shortness of breath all maintained strong internal correlations between physical activity levels, as well.

The authors noted that the NTDT-PRO showed robust reproducibility and reliability, was developed and screened in multiple languages and across multiple global regions, and may have clinical applications worldwide.

Reference

1. Taher A, Cappellini MD, Viprakasit V, et al. Validation of a patient-reported outcomes symptom measure for patients with nontransfusion-dependent thalassemia (NTDT-PRO^©) [published online November 5, 2018]. Am J Hematol. doi: 10.1002/ajh.25344