TY - JOUR
T1 - Key4OI Recommendations for Lung Function Guidance in Osteogenesis Imperfecta
T2 - Based on an Internationally Performed Comprehensive International Consortium for Health Outcomes Measurement Procedure
AU - Chaney, Hollis
AU - Mekking, Dagmar
AU - de Bakker, Danielle
AU - Beeri, Eliezer
AU - Eekhoff, E. Marelise W.
AU - Franken, Anton
AU - Kamp, Otto
AU - Micha, Dimitra
AU - Barreiros, C. u
AU - Tomlow, Ben
AU - van den Aardweg, Joost G.
AU - LoMauro, Antonella
AU - Folkestad, Lars
N1 - Funding Information:
Other contributions: The authors gratefully acknowledge Dagmar Mekking and the Care4BrittleBones Foundation for initiating and supporting the work on this project. The Key4OI is owned by Foundation Care4BrittleBones on behalf of everyone who contributed to its development. Additional information: The e-Appendix is available online under “Supplementary Data.”
Publisher Copyright:
© 2023 American College of Chest Physicians
PY - 2023/5/1
Y1 - 2023/5/1
N2 - Introduction: Pulmonary involvement in Osteogenesis Imperfecta (OI) can be severe but may be overlooked in milder cases. The Care4BrittleBones Foundation initiated this project to develop a set of global outcome measures focusing on respiratory-related issues in patients with OI. The objective was to reach an international consensus for a standardized set of outcomes and associated measuring instruments for the pulmonary care of individuals with OI. Based on the initial tests and questionnaires, we suggest parameters for when pulmonologists should seek guidance from the growing literature on OI pulmonary care and/or recognized experts in the field. Study Design and Methods: The project team consisted of a multidisciplinary mix of 12 people from six countries, including an OI patient representative, and facilitated by the Care4BrittleBones Foundation director. The International Consortium for Health Outcomes Measurement (ICHOM) process was followed, which includes the Delphi method, used to collect the opinions of the expert team. Patient input was present in each meeting due to the inclusion of a patient representative. In addition, online focus groups were held. They consisted of adults with OI from different countries, and they determined which questions matter the most to the OI community worldwide. Results: After three Delphi rounds, the expert team reached a consensus on the final set of measuring instruments, which included pulmonary function testing and patient self-reporting of symptoms related to breathing and sleep. Two questionnaires were decided upon: St. George's Respiratory Questionnaire (shortened version) and four questions regarding sleep. Patients should be screened for a history of pneumonia. Advanced testing for select patients by a pulmonologist would include further pulmonary function tests and a chest radiograph. Conclusions: A standardized set of outcome measures related to pulmonary care of individuals with OI was determined based on what is important to both experts and patients. This included patient-reported outcome measures and basic pulmonary function testing. Using these outcome measures, it can be determined which patients are at high risk for pulmonary complications.
AB - Introduction: Pulmonary involvement in Osteogenesis Imperfecta (OI) can be severe but may be overlooked in milder cases. The Care4BrittleBones Foundation initiated this project to develop a set of global outcome measures focusing on respiratory-related issues in patients with OI. The objective was to reach an international consensus for a standardized set of outcomes and associated measuring instruments for the pulmonary care of individuals with OI. Based on the initial tests and questionnaires, we suggest parameters for when pulmonologists should seek guidance from the growing literature on OI pulmonary care and/or recognized experts in the field. Study Design and Methods: The project team consisted of a multidisciplinary mix of 12 people from six countries, including an OI patient representative, and facilitated by the Care4BrittleBones Foundation director. The International Consortium for Health Outcomes Measurement (ICHOM) process was followed, which includes the Delphi method, used to collect the opinions of the expert team. Patient input was present in each meeting due to the inclusion of a patient representative. In addition, online focus groups were held. They consisted of adults with OI from different countries, and they determined which questions matter the most to the OI community worldwide. Results: After three Delphi rounds, the expert team reached a consensus on the final set of measuring instruments, which included pulmonary function testing and patient self-reporting of symptoms related to breathing and sleep. Two questionnaires were decided upon: St. George's Respiratory Questionnaire (shortened version) and four questions regarding sleep. Patients should be screened for a history of pneumonia. Advanced testing for select patients by a pulmonologist would include further pulmonary function tests and a chest radiograph. Conclusions: A standardized set of outcome measures related to pulmonary care of individuals with OI was determined based on what is important to both experts and patients. This included patient-reported outcome measures and basic pulmonary function testing. Using these outcome measures, it can be determined which patients are at high risk for pulmonary complications.
KW - Osteogenesis Imperfecta
KW - measuring instruments
KW - patient-reported outcomes
KW - pulmonary function
UR - http://www.scopus.com/inward/record.url?scp=85153624192&partnerID=8YFLogxK
U2 - 10.1016/j.chest.2022.12.047
DO - 10.1016/j.chest.2022.12.047
M3 - Article
C2 - 36640996
SN - 0012-3692
VL - 163
SP - 1201
EP - 1213
JO - Chest
JF - Chest
IS - 5
ER -