OBJECTIVES:

To define a Standard Set of outcomes and the most appropriate instruments to measure them for managing newly diagnosed multiple myeloma (MM) patients.

METHODS:

A Scientific Committee led the project. A literature review was performed to identify MM clinical outcomes, Patient Reported Outcomes (PROs) and the instruments to measure them. Later five discussion groups facilitated the design of 2-round Delphi questionnaire.

Delphi panellist (haematologists, hospital pharmacists and patients) were identified by the Scientific Committee, the Spanish Program of Haematology Treatments Foundation, the Spanish Society of Hospital Pharmacies and the Spanish Community of MM Patients. Panellist’s perception about outcomes’ suitability and feasibility of use were assessed on a 7-point Likert scale. Outcomes categories included survival and disease control, treatment complications PROs, patient reported experiences and patients’ basal characteristics. Consensus was reached when at least 75% of the respondents reached agreement or disagreement.

RESULTS:

Fifty-one and forty-five panellists participated in the first and second Delphi-round, respectively. Consensus was reached to use overall survival, progression-free survival, minimal residual disease and treatment response to assess survival and disease control. Panellists agreed to measure health-related quality of life, pain, performance status, fatigue, psychosocial status, symptoms, self-perception on body image, sexuality, and preferences/satisfaction. However, panellist did not reached consensus about the feasibility of assessing in routine practice psychosocial status, symptoms, self-perception on body image and sexuality. Consensus was reached to collect PROs through the EORTC-QLQ-C30 questionnaire, three items from EORTC-QLQ-MY20 and EORTC-QLQ-BR23, pain visual analogue scale, Morisky-Green and ad-hoc questions about patients’ preferences/satisfaction. Basal characteristics to be collected included age, gender, ethnicity, family history, international staging system, MM complications, comorbidities and treatment initiated.

CONCLUSIONS:

A consensual Standard Set of outcomes for managing newly diagnosed MM patients has been defined. The feasibility of its implementation in routine practice will be assesses in a future pilot study.