Measurement instruments for the core outcome set of congenital melanocytic naevi and an assessment of the measurement properties according to COSMIN: a systematic review.

Background: Congenital melanocytic naevi (CMN) can impact on patients' lives due to their appearance and the risk they carry of neurological complications or melanoma development. The development of a core outcome set (COS) will allow standardised reporting and enable comparison of outcomes. This will help to improve guidelines. In previous research, relevant stakeholders reached a consensus over which core outcomes should be measured in any future care or research. The next step of the COS development is to select the appropriate measurement instruments. Aim: Step 1: to update a systematic review identifying all core outcomes and measurement instruments available for CMN. Step 2: to evaluate the measurement properties of the instruments for the core outcomes. Methods: This study was registered in PROSPERO and performed according to the PRISMA checklist. Step 1 includes a literature search in EMBASE (Ovid), PubMed and the Cochrane Library to identify core outcomes and instruments previously used in research of CMN. Step 2 yields a systematic search for studies on the measurement properties of instruments that were either developed or validated for CMN, including a methodological quality assessment following the COSMIN methodology. Results: Step 1 included twenty-nine studies. Step 2 yielded two studies, investigating two quality of life measurement instruments. Conclusion: Step 1 provided an overview of outcomes and instruments used for CMN. Step 2 showed that additional research on measurement properties is needed to evaluate which instruments can be used for the COS of CMN. This study informs the instrument selection and/or development of new instruments.

Domains and outcomes of the core outcome set of congenital melanocytic naevi for clinical practice and research (the OCOMEN project): part 2.

BACKGROUND: Congenital melanocytic naevi (CMN) can have a great impact on patients' lives owing to perceived stigmatization, and the risk of melanoma development and neurological complications. Development of a core outcome set (COS) for care and research in CMN will allow standard reporting of outcomes. This will enable comparison of outcomes, allowing professionals to offer advice about the best management options. In previous research, stakeholders (patients, parents and professionals) reached consensus on the core domains of the COS. To select the appropriate measurement instruments, the domains should be specified by outcomes. OBJECTIVES: To reach consensus on the specific core outcomes describing the core domains pertaining to clinical care and research in CMN. METHODS: A list of provisional outcomes (obtained earlier) was critically reviewed by the Outcomes for COngenital MElanocytic Naevi (OCOMEN) research team and by relevant stakeholders through an online questionnaire, to refine this list and provide clear definitions for every outcome. When needed, discussion with individual participants was undertaken over the telephone or by email. During an online consensus meeting, stakeholders discussed the inclusion of potential outcomes. After the meeting, participants voted in two rounds for the inclusion of outcomes. RESULTS: Forty-four stakeholders from 19 countries participated. Nine core outcomes were included in the COS relative to clinical care and 10 core outcomes for research. CONCLUSIONS: These core outcomes will enable standard reporting in future care and research of CMN. This study facilitates the next step of COS development: selecting the appropriate measurement instruments for every outcome.

Development of an international core domain set for medium, large and giant congenital melanocytic naevi as a first step towards a core outcome set for clinical practice and research.

BACKGROUND: Medium, large and giant congenital melanocytic naevi (CMN) can impose a psychosocial burden on patients and families, and are associated with increased risk of developing melanoma or neurological symptoms. Lack of consensus on what outcomes to measure makes it difficult to advise patients and families about treatment and to set up best practice for CMN. OBJECTIVES: Fostering consensus among patient representatives and professionals, we aim to develop a core outcome set, defined as the minimum set of outcomes to measure and report in care and all clinical trials of a specific health condition. We focused on the 'what to measure' aspect, the so-called core domain set (CDS), following the COMET and CS-COUSIN guidelines. METHODS: We conducted a systematic review to identify outcomes reported in the literature. Focus groups with patient representatives identified patient-reported outcomes. All these outcomes were classified into domains. Through e-Delphi surveys, 144 stakeholders from 27 countries iteratively rated the importance of domains and outcomes. An online consensus meeting attended by seven patient representatives and seven professionals finalized the CDS. RESULTS: We reached consensus on six domains, four of which were applied to both care and research: 'quality of life', 'neoplasms', 'nervous system' and 'anatomy of skin'. 'Adverse events' was specific to care and 'pathology' to research. CONCLUSIONS: We have developed a CDS for medium-to-giant CMN. Its application in reporting care and research of CMN will facilitate treatment comparisons. The next step will be to reach consensus on the specific outcomes for each of the domains and what instruments should be used to measure these domains and outcomes.

Outcomes and measurement instruments used in congenital melanocytic naevi research: A systematic review.

BACKGROUND: Congenital melanocytic naevi (CMN) can have a great impact on patients' lives due to the remarkable appearance and the risk of developing melanoma and neurocutaneous melanosis. Comparison of treatment efficacy is currently hindered by the lack of standard and uniform outcome reporting; this impedes guidance on optimal management policy. To address this, we aim to perform the first step in developing a core outcome set. With this systematic review, we identified a list of domains, outcomes (including patient reported outcomes) and outcome measurement instruments used in CMN research. METHODS: The review was registered in PROSPERO, registration number CRD42018095235. A search was conducted in EMBASE (Ovid), PubMed and the Cochrane Library from 2006 to January 2019. Studies with 10 or more patients, with all sizes of CMN and reporting outcomes on interventional and conservative management were included. RESULTS: A total of 1,285 individual studies was found; 63 studies were included. We extracted 57 different outcomes and 34 outcome measurement instruments showing large heterogeneity. Patient-reported outcomes were included in 38% of studies. Few outcome measurement instruments were described. Moreover, none of the studies reported that the used instruments were ever validated in a CMN population. CONCLUSION: Heterogeneity exists in outcomes and instruments used in CMN research. The development of a core outcome set may reduce this heterogeneity in future research, thereby enabling treatment comparison and eventually facilitating guidance on management. Furthermore, this overview demonstrates a need for the use and validation of (patient reported) outcome measurement instruments for CMN.

Protocol for the development of core set of domains of the core outcome set for patients with congenital melanocytic naevi (OCOMEN project).

BACKGROUND: Having large congenital melanocytic naevi (CMN) is associated with a psychosocial burden on patients and their parents because of its remarkable appearance and the extra care it may require. Large CMN also pose an increased risk of malignant melanoma or neurocutaneous melanosis. There is a lack of international consensus on what important outcome domains to measure in relation to treatment. This makes it difficult to compare options, to properly inform patients and their parents, and to set up treatment policy for CMN. Therefore, we aim to develop a core outcome set (COS), i.e. the minimum set of outcomes that are recommended to be measured and reported in all clinical trials of a specific health condition. This COS can be used in the follow-up of CMN patients with or without treatment, in clinical research and practice. METHODS: In the Outcomes for Congenital Melanocytic Nevi (OCOMEN) projects, we follow the recommendations from the Core Outcome Measures in Effectiveness Trials (COMET) initiative and the Cochrane Skin Core Outcomes Set Initiative (CS-COUSIN). This project entails the following: (i) a systematic review to identify the previous reported outcomes in literature; (ii) focus groups with national and international patients and parents to identify patient-important outcomes; (iii) classification of outcomes into outcome domains; (iv) e-Delphi surveys in which stakeholders (patients/parents and professionals) can rate the importance of domains and outcomes; and (v) an online consensus meeting to finalize the core outcome domains of the COS. RESULTS: The results will be disseminated by means of publication in a leading journal and presentations in international meetings or conferences. We engage international experts in CMN, both patients and professionals, to ensure the international utility and applicability of the COS.