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Categorizing factors of adherence to parenteral treatment in growth hormone deficiencies and hemophilia: What should be the targets for future research?

Authors Sultan S, El-Hourani M, Rondeau É, Garnier N

Received 14 July 2018

Accepted for publication 16 September 2018

Published 8 October 2018 Volume 2018:12 Pages 2039—2063


Checked for plagiarism Yes

Review by Single anonymous peer review

Peer reviewer comments 2

Editor who approved publication: Dr Johnny Chen

Serge Sultan,1–3 Mira El-Hourani,3 Émélie Rondeau,1 Nicolas Garnier4

1Department of Hematology-Oncology, CHU Sainte-Justine, Montréal, Québec, Canada; 2Department of Pediatrics, Université de Montréal, Montréal, Québec, Canada; 3Department of Psychology, Université de Montréal, Montréal, Québec, Canada; 4Pfizer Canada Inc, Rare Disease Unit, Medical Affairs, Kirkland, Québec, Canada

Abstract: Adherence to treatment regimens in growth hormone dysregulations and hemophilia is related to better outcome and fewer complications over time. Subcutaneous growth hormone injection and intravenous blood factor replacement therapies are parenteral treatments with a comparable regimen calling for similar behavioral processes. Although we have lists of possible factors influencing adherence in these conditions, the evidence is scattered. The objective of this study was to systematically review empirical studies linking factors of adherence with measures of adherence. To categorize the factors, we used a taxonomy from the diabetes literature. We used four major electronic databases to identify articles. We synthesized 27 articles dated 2011–2017 corresponding to inclusion criteria. Results showed a consistent proportion of 20%–25% participants with adherent issues. Strong arguments pointed to the transition to self-care in pediatrics as a vulnerability period (7/27 reports). We found the domains of individual factors (<30% reports), relational factors (<13%), health care (<30%), to be understudied in comparison with that of demographic or clinical context (>74%), and practical issues (>37%). The results suggest that future research should focus on modifiable factors of adherence, with appropriate measurement and intervention strategies. One central methodological limitation of reviewed reports was the lack of longitudinal designs, and the quasi absence of behavioral trial targeting modifiable factors of adherence. A new research agenda should be set in these rare diseases as higher adherence should translate into improved outcome and better quality of life for patients and their families.

adherence, factors, predictors, classification, growth hormone, hemophilia

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