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. 2021 Sep 28;5(1):102.
doi: 10.1186/s41687-021-00376-0.

A novel patient-reported outcome instrument assessing the symptoms of paroxysmal nocturnal hemoglobinuria, the PNH-SQ

R Paola Daly  1 , Jessica J Jalbert  2 , Shannon Keith  1 , Tara Symonds  3 , Jamile Shammo  4
Affiliations

A novel patient-reported outcome instrument assessing the symptoms of paroxysmal nocturnal hemoglobinuria, the PNH-SQ

R Paola Daly et al. J Patient Rep Outcomes. .

Abstract

Background: Patient-reported outcome measures (PROs) used to measure symptoms of patients with paroxysmal nocturnal hemoglobinuria (PNH) in trials do not measure PNH symptoms comprehensively and do not assess daily fluctuations in symptoms. Following a literature review and consultation with a PNH expert, we drafted the PNH Symptom Questionnaire (PNH-SQ) and a patient-centric conceptual model of PNH symptoms and impacts. We then interviewed 15 patients with PNH to assess comprehensiveness of symptom capture from the patient perspective and to cognitively debrief the PNH-SQ. Patient interview data were also used to finalize the PNH conceptual model.

Results: Participants mentioned 27 signs or symptoms of PNH spontaneously or after being probed; 93% reported experiencing ≥ 1 PNH symptom. Concept saturation was reached for all PNH symptoms. Further, interviews confirmed the instrument captured the most common PNH symptoms, including fatigue (87%), abdominal pain (60%), and difficulty swallowing (47%), with fatigue ranked as the most bothersome symptom. The interviews demonstrated that participants understood the items of the PNH-SQ (90-100%); considered the symptoms relevant (> 50- > 90%); the recall period appropriate (> 80-100%); and the response options suitable (> 80-100%). Participants also suggested changes regarding item redundancy and relevance; this feedback was used to finalize the instrument.

Conclusions: The finalized PNH-SQ assesses the presence and severity of 10 symptoms-abdominal pain, chest discomfort, difficulty sleeping, difficulty swallowing, difficulty thinking clearly, fatigue, headache, muscle weakness, pain in the legs or back, and shortness of breath-over 24 h. The PNH-SQ is a content-valid questionnaire suitable for assessing daily symptom presence and severity in PNH clinical trials.

Keywords: PNH; PRO; QoL; Symptom.

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Conflict of interest statement

R.P.D. was an employee of Clinical Outcomes Solutions; J.J.J. is an employee of Regeneron Pharmaceuticals, Inc.; S.K. and T.S. are employees of Clinical Outcomes Solutions; J.S. has the following disclosures: Regeneron Pharmaceuticals, Inc.: Consultancy; Incyte: Consultancy, Honoraria, Research Funding, Speakers Bureau; CTI Pharma: Research Funding; Onconova: Research Funding; Alexion: Consultancy, Honoraria, Research Funding, Speakers Bureau; Celgene: Consultancy, Honoraria, Research Funding, Speakers Bureau; Apellis: Membership on an entity's Board of Directors or advisory committees; Otsuka: Consultancy, Honoraria; Novartis: Consultancy, Honoraria; Astex Pharma: Research Funding; Sanofi: Consultancy, Honoraria, Speakers Bureau.

Figures

Fig. 1
Fig. 1
Literature review search terms
Fig. 2
Fig. 2
Conceptual model of PNH. PNH, paroxysmal nocturnal hemoglobinuria
Fig. 3
Fig. 3
Frequency of PNH symptoms experienced. PNH paroxysmal nocturnal hemoglobinuria
See this image and copyright information in PMC

References

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