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Which Functional Classification Scale is Optimal for Children with Pulmonary Hypertension (PAH)?

ABSTRACT: Functional status assessed by the WHO-FC scale derived from adults is a known prognostic factor for pulmonary hypertension. Data on the usefulness of the Panama-FC scale in assessing children with pulmonary hypertension are limited. The study was performed to compare functional status results (WHO-FC and Panama-FC) and to assess the usefulness of these scales in various clinical situations. The reliability of the Panama-FC questionnaire method for facilitating patient evaluation was also examined. 26 functional status assessments (7 in disease progression/after treatment intensification) in both scales were analyzed in 19 patients with PAH confirmed in RHC. WHO-FC, Panama-FC scales, and questionnaire-based on Panama-FC were conducted independently by three different physicians. Results of assessments were compared with each other and with 6MWD, NTproBNP level, and echo parameters (TAPSE, RV/LV ratio). The Panama-FC scale results obtained using the medical interview method and questionnaire did not differ. Both WHO-FC and Panama-FC classes well-reflected disease advancement confirmed by non-invasive parameters (NTproBNP, 6MWD, TAPSE, RV/LV ratio). Differences between grading the class in both scales were observed: 5pts were classified to II (Panama-FC) vs I (WHO-FC), 2pts were in lower risk group in WHO-FC (II) vs Panama (IIIa). Worsening or improvement after treatment intensification in functional status in both scales was connected with the significant change of NTproBNP level. The 6-min walking distance did not change. TAPSE, RV/LV ratio changed significantly in 3pts with IPAH, accordingly to change in WHO-FC and Panama-FC. WHO-FC and Panama-FC well reflect the disease advancement. The questionnaire method simplified the use of the Panama-FC scale. The Panama-FC scale appears to be better for assessing functional status during long-term follow-up, while the WHO-FC scale was more useful in short-term treatment monitoring.

SUBMITTER: Migdal A

PROVIDER: S-EPMC7695665 | biostudies-literature | 2020 Dec

REPOSITORIES: biostudies-literature

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Which Functional Classification Scale is Optimal for Children with Pulmonary Hypertension (PAH)?

Migdał Anna A Żuk Małgorzata M Jagiełłowicz-Kowalska Dorota D Powichrowska Zuzanna Z Brzezińska-Rajszys Grażyna G

Pediatric cardiology 20200809 8

Functional status assessed by the WHO-FC scale derived from adults is a known prognostic factor for pulmonary hypertension. Data on the usefulness of the Panama-FC scale in assessing children with pulmonary hypertension are limited. The study was performed to compare functional status results (WHO-FC and Panama-FC) and to assess the usefulness of these scales in various clinical situations. The reliability of the Panama-FC questionnaire method for facilitating patient evaluation was also examine ...[more]

PMID: 32772124

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Project description:Regulators and clinical experts increasingly recognize the importance of incorporating patient-reported outcomes (PROs) in clinical studies of therapies for pulmonary arterial hypertension (PAH). No PAH-specific instruments have been developed to date in accordance with the 2009 FDA guidance for the development of PROs as endpoints in clinical trials. A qualitative research study was conducted to develop a new instrument assessing PAH symptoms and their impacts following the FDA PRO guidance.A cross-sectional study was conducted at 5 centers in the US in symptomatic PAH patients aged 18-80 years. Concept elicitation was based on 5 focus group discussions, after which saturation of emergent concepts was reached. A PRO instrument for PAH symptoms and their impacts was drafted. To assess the appropriateness of items, instructions, response options, and recall periods, 2 rounds of one-on-one cognitive interviews were conducted, with instrument revisions following each round. Additional interviews tested the usability of an electronic version (ePRO). PRO development considered input from an international Steering Committee, and translatability and lexibility assessments.Focus groups comprised 25 patients (5 per group); 20 additional patients participated in cognitive interviews (10 per round); and 10 participated in usability interviews. Participants had a mean?±?SD age of 53.1?±?15.8 years, were predominantly female (93 %), and were diverse in race/ethnicity, WHO functional class (FC I/II: 56 %, III/IV: 44 %), and PAH etiology (idiopathic: 56 %, familial: 2 %, associated: 42 %). The draft PRO instrument (PAH-SYMPACT®) was found to be clear, comprehensive, and relevant to PAH patients in cognitive interviews. Items were organized in a draft conceptual framework with 16 symptom items in 4 domains (respiratory symptoms, tiredness, cardiovascular symptoms, other symptoms) and 25 impact items in 5 domains (physical activities, daily activities, social impact, cognition, emotional impact). The recall period is the past 24 h for symptoms, and the past 7 days for impacts.The PAH-SYMPACT® was shown to capture symptoms and their impacts relevant to PAH patients, demonstrating content saturation, concept validity, and ePRO usability. Final content and psychometric validation of the instrument will be based on the results of an ongoing Phase IIIb clinical trial in PAH patients.

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Which Functional Classification Scale is Optimal for Children with Pulmonary Hypertension (PAH)?

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Which Functional Classification Scale is Optimal for Children with Pulmonary Hypertension (PAH)?

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