Randomized clinical trials in periodontology: focus on outcomes selection

PANNUTI, Claudio Mendes; COSTA, Fernando Oliveira; SOUZA, Nathalia Vilela; RETAMAL-VALDES, Belen; COSTA, Amanda Almeida; SUSIN, Cristiano; FERES, Magda

doi:10.1590/1807-3107bor-2021.vol35.0100

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Critical Review, Periodontology • Braz. oral. res. 35 (Supp 2) • 2021 • https://doi.org/10.1590/1807-3107bor-2021.vol35.0100 linkcopy

Randomized clinical trials in periodontology: focus on outcomes selection

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Abstract

Randomized clinical trials (RCTs) are human studies carried out to compare different treatments or interventions, and their results are used to support clinical decision-making and improve patient care. Herein, the aim of this study was to review the selection process of study outcomes in periodontology. Primary outcomes should draw the main conclusions of the study, whereas secondary outcomes should only be used to help explain the main findings and generate future research hypothesis. Outcomes are classified as clinically relevant (CROs) or surrogate outcomes. CROs – the first option for primary outcome variables - should convey not only substantial health benefits, but also be deemed important by patients. In periodontology, tooth loss/retention and oral health–related quality of life (OHRQoL) are examples of CROs. While tooth loss has main limitations as a primary outcome, emerging evidence suggest that patient-reported outcome measures (PROMs) can accurately detect OHRQoL following periodontal therapy. When CROs cannot be assessed, validated surrogate outcomes can be used as proxies. Primary outcome variables should reflect a treatment endpoint at the patient level that can be easily used to inform decision-making in daily practice. These outcomes should allow the implementation of a treat-to-target concept in which the intervention can be clearly judged against a prespecified treatment target. Recently, the presence of at most 4 sites with periodontal probing depth ≥5 mm post-treatment was suggested as an effective endpoint for periodontal trials. In perspective, a combination of validated clinical parameters and PROMs will provide a more comprehensive assessment of periodontal treatments.

Clinical Trial; Treatment Outcome

Author (year)	Intervention and sample	Time evaluation	PROM evaluation	Main results
Santuchi et al., 2016²¹	Scaling and root planning (n = 45) and full-mouth debridement (n = 45)	Baseline, 1 and 6 months	OIDP	Both groups showed significant improvement on clinical parameters and OIDP; no significant differences between groups.
Åslund et al., 2008²⁴	Non-surgical therapy using a piezo-ceramic device (n = 30) or curets (n = 29) in individuals with mild to moderate periodontitis	Baseline, treatment, and 1, 4, and 8 weeks	OHQoL-UK	Both groups showed improvements in clinical parameters, pain scores and QoL measures
Agado et al., 2012²⁵	Utrasonic debridement (n = 10), hand instrumentation (n = 10), and a control group (n = 10) in chronic obstructive pulmonary disease patients	Baseline and 4 weeks post-treatment	SGRQ	Total SGRQ-A scores decreased slightly for all groups with no significant difference among groups and no significant interaction
Santuchi et al., 2015²⁶	Scaling and root planning per quadrant (n = 37) and one-stage full-mouth disinfection (n = 41)	Baseline and 6 months	DFS, DAS and VAS	Patients with higher PROs showed worse clinical parameters before and after treatment. After both treatments, fear and anxiety decreased with no significant differences between groups. No significant differences in pain scores between groups
Cortelli et al., 2018²⁷	Ultrasonic debridement in combination with essential-oils in diabetic (n = 30) and non-diabetic individuals (n = 30), or placebo mouthwash in diabetic (n = 30) and non-diabetic individuals (n = 30)	Baseline and 3 months	OHQoL-UK	Combined treatment with EO provided OHQoL improvements in both systemic conditions, but not in placebo groups
Musskopf et al., 2018²⁸	Pregnant women receiving scaling and root planning/maintenance (n = 96), or supragingival scaling and polishing (n = 114)	Before and after treatment	OHIP-14	Both groups showed significant reduction in OHIP-14 scores; control group had significantly higher odds of worsening their OHIP-14 scores and their perception of oral conditions than test group
Zhou et al., 2019²⁹	60 CPOD individuals: (n = 20) scaling and root planning treatment, (n = 20) supragingival scaling treatment and (n = 20) oral hygiene instructions only with no periodontal treatment	Baseline, 1 and 2 years	SGRQ	The impacts scores of two treatment groups were significantly lower than control group at 2-year follow-up

Type of outcome	Definition	Examples
Clinically relevant outcomes (CROs)	Outcomes that directly measure how a patient feels, functions or survives	Death, number of hospitalizations, tooth loss, oral health-related quality of life
Patient-reported outcome measures (PROMs)	Measurements of any aspect of a patient’s health status that come directly from the patient. PROMs are considered to be CROs.	Pain/discomfort, self-assessment of function and esthetics, oral health-related quality of life
Surrogate outcomes	Outcomes that are not themselves a direct measurement of the clinical endpoint. Rather, they are supposed to be a proxy for the true CRO	Gingival crevicular fluid levels of interleukin-1β, radiographic alveolar bone loss, clinical attachment gain
Primary outcome	The pre-specified outcome considered to be of greatest importance to the study; study design and sample size calculations are based on the primary outcome	Depending on the objective of the study a given outcome can be primary or secondary.
Secondary outcomes	All other outcomes in the study. The trial is not designed to evaluate them, so they are exploratory in nature	E.g.: pain assessed using a visual analog scale can be a primary outcome in an RCT testing an analgesic and secondary in a RCT comparing two root coverage surgical techniques

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Sociedade Brasileira de Pesquisa Odontológica - SBPqO Av. Prof. Lineu Prestes, 2227, 05508-000 São Paulo SP - Brazil, Tel. (55 11) 3044-2393/(55 11) 9-7557-1244 - São Paulo - SP - Brazil
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[TFN1] OHQoL-UK: United Kingdom OHQoL questionnaire; SGRQ: St. George’s Respiratory Questionnaire; DFS: Dental Fear Survey; DAS: Dental Anxiety Scale VAS: Visual Analogue Scale; OIDP: Oral Impacts on Daily Performance; OHIP-14: Oral Health Impact Profile-14.