Development of a patient ‐reported outcomes symptom measure for patients with nontransfusion‐dependent thalassemia (NTDT‐PRO©)

This study aimed to develop a new, US Food and Drug Administration (FDA)‐compliant PRO of chronic anemia symptoms, the NTDT‐PRO© tool, for use in patients with NTDT. Participants had a median age of 36  years (range, 18‐47) and 60% were female. The initial development of the NTDT‐PRO tool involved concept‐elicitation interviews with 25 patients from 3 centers (in Lebanon, Greece, and Canada); subsequent interview discussions and clinical input resulted in the generation of 9 items for incl usion in the draft NTDT‐PRO. Following a round of cognitive interviews involving 21 patients from 2 centers (in Lebanon and Greece), 4 items (Pain, Headaches, Ability to Concentrate, and Paleness) were removed from the draft NTDT‐PRO. The final NTDT‐PRO comprises 6 items that measure Tiredness , Weakness, and Shortness of Breath, with or without Physical Activity. The NTDT‐PRO is a new disease‐specific HRQoL tool for patients with NTDT, developed using a thorough methodology based on FDA 2009 PRO development guidelines.
Source: American Journal of Hematology - Category: Hematology Authors: Tags: RESEARCH ARTICLE Source Type: research