OBJECTIVES: Collect quantitative data on drivers of patient treatment preferences in paroxysmal nocturnal haemoglobinuria (PNH).

METHODS: PNH patients (n=156, U.S., n=117, EU and Canada n=39) completed a questionnaire ranking attractiveness of PNH treatment aspects, including product and clinical features, considering available treatments. A Q-methodology was used to collect the data and utility scores were computed as average ranks, on a -4 to +4 scale.

RESULTS: Fatigue levels and blood transfusions, together with dosing frequency had the highest preference among patients. Unlikeliness to feel fatigued at all was preferred (+2.13) compared to somewhat fatigued (-0.21) or very much fatigued (-1.64). This correlated with fatigue symptoms reported by patients as impactful on quality of life and mobility. Treatments that meant no annual blood transfusions required were preferred (+1.97) vs requirement of 1, 2, 3 or 4+ transfusions annually while taking the drug (-0.61, -1.46, -2.02 and -2.74 respectively). Administration every eight weeks was most valued (+2.62), vs every two weeks (+0.69), weekly (-0.38) or twice per week (-1.40). Route of administration was less impactful (+0.07 for subcutaneous pump; +0.08 for subcutaneous auto-injector; +0.44 for intravenous), however, aspects like mobility during administration (+1.74) and ability to self-administer at home or work (+1.17) were preferred. A needle hidden during administration was valued (+0.48) vs one visible (-0.02) or larger (+0.08). Scores were directionally similar for patients in Europe and Canada vs U.S., but less emphasis was given to no blood transfusion requirement (+1.31 and +2.19, respectively) and no fatigue (+1.97 and +2.19, respectively); reduced dosing (+2.77 and +2.57, respectively) and self-administration (+1.56 and +1.03, respectively) had a slightly importance outside of U.S.

CONCLUSIONS: Results suggest that the most important PNH treatment attributes are improvement in fatigue and reduced number of blood transfusions required, along with less frequent dosing. Mobility during administration, self-administration and non-visible needles are also preferred.