TY - JOUR
T1 - Facing the Realities of Pragmatic Design Choices in Environmental Health Studies
T2 - Experiences from the Household Air Pollution Intervention Network Trial
AU - Checkley, William
AU - Hossen, Shakir
AU - Rosa, Ghislaine
AU - Thompson, Lisa M.
AU - McCracken, John P.
AU - Diaz-Artiga, Anaite
AU - Balakrishnan, Kalpana
AU - Simkovich, Suzanne M.
AU - Underhill, Lindsay J.
AU - Nicolaou, Laura
AU - Hartinger, Stella M.
AU - Davila-Roman, Victor G.
AU - Kirby, Miles A.
AU - Clasen, Thomas F.
AU - Rosenthal, Joshua
AU - Peel, Jennifer L.
N1 - Funding Information:
Funding: This study is funded by the U.S. National Institutes of Health (cooperative agreement UM1HL134590; Multiple Principal Investigators: Checkley, Clasen, Peel) in collaboration with the Bill & Melinda Gates Foundation (OPP1131279). Participating National Institutes of Health organizations include the National Heart, Lung and Blood Institute, National Institute of Environmental Health Sciences, National Cancer Institute, National Institute of Child Health and Human Development, Fogarty International Center, and the National Institutes of Health Common Fund. Suzanne M. Simkovich was supported by funding from the National Heart, Lung and Blood Institute (F32HL143909, K12HL137942). Lindsay J. Underhill was supported by the UMJT Fogarty Global Health Fellowship Training Grant (D43TW009340; Multiple Principal Investigators: Buekens, Check-ley, Chi, Kondwani) funded by the United States National Institutes of Health through the following Institutes and Centers: Fogarty International Center; National Institute of Neurological Disorders and Stroke; National Institute of Mental Health; National Heart, Lung, and Blood Institute; and, the National Institute of Environmental Health Sciences.
Funding Information:
Acknowledgments: This Household Air Pollution Intervention Network was funded by the United States National Institutes of Health (UM1HL134590; MPI: Checkley, Clasen, Peel). A multidisciplinary, independent Data and Safety Monitoring Board (DSMB) appointed by the National Heart, Lung, and Blood Institute (NHLBI) monitors the quality of the data and protects the safety of patients enrolled in the HAPIN trial. NHLBI DSMB: Nancy R. Cook; Stephen Hecht; Catherine Karr; Katie H. Kavounis; Dong-Yun Kim; Joseph Millum; Lora A. Reineck; Nalini Sathiakumar; Paul K. Whelton; Gail G. Weinmann. Program Coordination: Gail Rodgers, Bill & Melinda Gates Foundation; Claudia L. Thompson, National Institute of Environmental Health Science (NIEHS); Mark J. Parascandola, M.P.H., National Cancer Institute (NCI); Danuta M. Krotoski, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); Joshua P. Rosenthal, Fogarty International Center (FIC); Conception R. Nierras, NIH Office of Strategic Coordination Common Fund; Antonello Punturieri and Barry S. Schmetter, B.S., National Heart, Lung, and Blood Institute (NHLBI). The findings and conclusions in this report are those of the authors and do not necessarily represent the official position of the U.S. National Institutes of Health or Department of Health and Human Services. HAPIN Investigators: Vigneswari Aravindalochanan, Kalpana Balakrishnan, Dana Boyd Barr, Vanessa Burrowes, Devan Campbell, Julia McPeek Campbell, Eduardo Canuz, Adly Castañaza, Howard Chang, William Checkley, Yunyun Chen, Marilú Chiang, Maggie L. Clark, Thomas Clasen, Rachel Craik, Mary Crocker, Victor Dávila-Román, Lisa de las Fuentes, Oscar De Léon, Anaité Diaz-Artiga, Ephrem Dusabimana, Lisa Elon, Juan Gabriel Espinoza, Irma Sayury Pineda Fuentes, Sarada Garg, Dina Goodman, Savannah Gupton, Meghan Hardison, Stella Hartinger, Steven A. Harvey, Mayari Hengstermann, Phabiola Herrera, Shakir Hossen, Penelope Howards, Lindsay Jaacks, Shirin Jabbarzadeh, Michael A. Johnson, Abigail Jones, Katherine Kearns, Miles Kirby, Jacob Kremer, Margaret Laws, Patricia M. Lenzen, Jiawen Liao, Amy Lovvorn, Fiona Majorin, Eric McCollum, John P. McCracken, Rachel M. Meyers, J. Jaime Miranda, Erick Mollinedo, Lawrence Moulton, Krishnendu Mukhopadhyay, Laura Nicolaou, Luke Naeher, Abidan Nambajimana, Florien Ndagijimana, Azhar Nizam, Jean de Dieu Ntivuguruzwa, Aris Papageorghiou, Jennifer Peel, Ricardo Piedrahita, Ajay Pillarisetti, Naveen Puttaswamy, Elisa Puzzolo, Ashlinn Quinn, Sarah Rajkumar, Usha Ramakrishnan, Davis Reardon, Ghislaine Rosa, Joshua Rosenthal, P. Barry Ryan, Zoe Sakas, Sankar Sambandam, Jeremy Sarnat, Suzanne Simkovich, Sheela Sinharoy, Kirk R. Smith, Kyle Steenland, Damien Swearing, Gurusamy Thangavel, Lisa M. Thompson, Ashley K. Toenjes, Lindsay Underhill, Jean Damascene Uwizeyimana, Viviane Valdes, Amit Verma, Lance Waller, Megan Warnock, Kendra Williams, Wenlu Ye, Bonnie Young.
Funding Information:
This study is funded by the U.S. National Institutes of Health (cooperative agreement UM1HL134590; Multiple Principal Investigators: Checkley, Clasen, Peel) in collaboration with the Bill & Melinda Gates Foundation (OPP1131279). Participating National Institutes of Health organizations include the National Heart, Lung and Blood Institute, National Institute of Environmental Health Sciences, National Cancer Institute, National Institute of Child Health and Human Development, Fogarty International Center, and the National Institutes of Health Common Fund. Suzanne M. Simkovich was supported by funding from the National Heart, Lung and Blood Institute (F32HL143909, K12HL137942). Lindsay J. Underhill was supported by the UMJT Fogarty Global Health Fellowship Training Grant (D43TW009340; Multiple Principal Investigators: Buekens, Checkley, Chi, Kondwani) funded by the United States National Institutes of Health through the following Institutes and Centers: Fogarty International Center; National Institute of Neurological Disorders and Stroke; National Institute of Mental Health; National Heart, Lung, and Blood Institute; and, the National Institute of Environmental Health Sciences.
Publisher Copyright:
© 2022 by the authors. Licensee MDPI, Basel, Switzerland.
PY - 2022/4/1
Y1 - 2022/4/1
N2 - Objective: Household Air Pollution Intervention Network (HAPIN) investigators tested a complex, non-pharmacological intervention in four low- and middle-income countries as a strategy to mitigate household air pollution and improve health outcomes across the lifespan. Intervention households received a liquefied petroleum gas (LPG) stove, continuous fuel delivery and regular behavioral reinforcements for 18 months, whereas controls were asked to continue with usual cooking practices. While HAPIN was designed as an explanatory trial to test the efficacy of the intervention on four primary outcomes, it introduced several pragmatic aspects in its design and conduct that resemble real-life conditions. We surveyed HAPIN investigators and asked them to rank what aspects of the design and conduct they considered were more pragmatic than explanatory. Methods: We used the revised Pragmatic Explanatory Continuum Indicator Summary (PRECIS-2) to survey investigators on the degree of pragmatism in nine domains of trial design and conduct using a five-point Likert rank scale from very explanatory (1) to very pragmatic (5). We invited 103 investigators. Participants were given educational material on PRECIS-2, including presentations, papers and examples that described the use and implementation of PRECIS-2. Results: Thirty-five investigators (mean age 42 years, 51% female) participated in the survey. Overall, only 17% ranked all domains as very explanatory, with an average (±SD) rank of 3.2 ± 1.4 across domains. Fewer than 20% of investigators ranked eligibility, recruitment or setting as very explanatory. In contrast, ≥50% of investigators ranked the trial organization, delivery and adherence of the intervention and follow-up as very/rather explanatory whereas ≤17% ranked them as rather/very pragmatic. Finally, <25% of investigators ranked the relevance of outcomes to participants and analysis as very/rather explanatory whereas ≥50% ranked then as rather/very pragmatic. In-country partners were more likely to rank domains as pragmatic when compared to investigators working in central coordination (average rank 3.2 vs. 2.8, respectively; Wilcoxon rank-sum p < 0.001). Conclusion: HAPIN investigators did not consider their efficacy trial to be rather/very explanatory and reported that some aspects of the design and conduct were executed under real-world conditions; however, they also did not consider the trial to be overly pragmatic. Our analysis underscores the importance of using standardized tools such as PRECIS-2 to guide early discussions among investigators in the design of environmental health trials attempting to measure efficacy.
AB - Objective: Household Air Pollution Intervention Network (HAPIN) investigators tested a complex, non-pharmacological intervention in four low- and middle-income countries as a strategy to mitigate household air pollution and improve health outcomes across the lifespan. Intervention households received a liquefied petroleum gas (LPG) stove, continuous fuel delivery and regular behavioral reinforcements for 18 months, whereas controls were asked to continue with usual cooking practices. While HAPIN was designed as an explanatory trial to test the efficacy of the intervention on four primary outcomes, it introduced several pragmatic aspects in its design and conduct that resemble real-life conditions. We surveyed HAPIN investigators and asked them to rank what aspects of the design and conduct they considered were more pragmatic than explanatory. Methods: We used the revised Pragmatic Explanatory Continuum Indicator Summary (PRECIS-2) to survey investigators on the degree of pragmatism in nine domains of trial design and conduct using a five-point Likert rank scale from very explanatory (1) to very pragmatic (5). We invited 103 investigators. Participants were given educational material on PRECIS-2, including presentations, papers and examples that described the use and implementation of PRECIS-2. Results: Thirty-five investigators (mean age 42 years, 51% female) participated in the survey. Overall, only 17% ranked all domains as very explanatory, with an average (±SD) rank of 3.2 ± 1.4 across domains. Fewer than 20% of investigators ranked eligibility, recruitment or setting as very explanatory. In contrast, ≥50% of investigators ranked the trial organization, delivery and adherence of the intervention and follow-up as very/rather explanatory whereas ≤17% ranked them as rather/very pragmatic. Finally, <25% of investigators ranked the relevance of outcomes to participants and analysis as very/rather explanatory whereas ≥50% ranked then as rather/very pragmatic. In-country partners were more likely to rank domains as pragmatic when compared to investigators working in central coordination (average rank 3.2 vs. 2.8, respectively; Wilcoxon rank-sum p < 0.001). Conclusion: HAPIN investigators did not consider their efficacy trial to be rather/very explanatory and reported that some aspects of the design and conduct were executed under real-world conditions; however, they also did not consider the trial to be overly pragmatic. Our analysis underscores the importance of using standardized tools such as PRECIS-2 to guide early discussions among investigators in the design of environmental health trials attempting to measure efficacy.
KW - Effectivness
KW - Efficacy
KW - Household air pollution
KW - Randomized trials
UR - http://www.scopus.com/inward/record.url?scp=85130219612&partnerID=8YFLogxK
U2 - 10.3390/ijerph19073790
DO - 10.3390/ijerph19073790
M3 - Article
C2 - 35409475
AN - SCOPUS:85130219612
VL - 19
JO - International Journal of Environmental Research and Public Health
JF - International Journal of Environmental Research and Public Health
SN - 1661-7827
IS - 7
M1 - 3790
ER -