Preliminary Development Of Patient-Reported Outcomes Instrument For Multiple Myeloma

Objective:To comprehensively understand the disease symptoms,mental state,treatment experience and social support of patients with multiple myeloma;to formulate Patient Report Outcome scales from the patient's perspective according to the principles and procedures regulated by the US Food and Drug Administration(FDA)Patients with myeloma report the outcome scale,which is used in clinical practice to truly reflect the patient's condition and provide reference for medical care decision-making and social assistance.Methods:(1)This study used literature review and expert discussion to construct the dimensions of the patient report outcome scales for patients with multiple myeloma.Through literature research,case review,questionnaire survey,and qualitative interview,comprehensively understand the disease symptoms,life quality,treatment experience,psychological state,social support of patients with multiple myeloma,and develop the pool of entries;(2)After two rounds of enquiries of 20 representative experts,according to the scores,to modify the entries according to expert opinions and the comprehensive evaluation of the research group and form initial Patient Report outcome scales of multiple myeloma(pre-survey scale).Results:(1)Literature research and expert discussion led to the framework of the reported outcome scale for patients with multiple myeloma,including 4 fields,physiological,psychological,therapeutic and social.(2)A review of 124 patients with multiple myeloma in the department of Hematology,Affiliated Drum Tower Hospital of Medical College in Nanjing University.the ratio of male to female was 1.43:1,the average age was 62.9 years old,the incidence of bone pain was 70.16%,the incidence of pathological fracture was 18.55%,the incidence of numbness and tingling in the hands and feet was 61.29%,the incidence of fatigue and weakness was 37.90%;(3)Questionnaire survey shows that psychological elasticity scores of MM patients is 56.24±16.22 points,patients were in poor mental state and gender,long-term residence,family monthly income,medical expenses payment methods,complications and social support were correlated to psychological status(P<0.05);(4)Qualitative interviews with MM patients were extracted out three major themes:poor physical experience(sensory abnormalities,numbness,pain,urinary incontinence),positive response(family support,increasing medical level,self-regulation),self-feeling burden(economic stress,recurrence)Short cycle and excessive psychological burden);(5)Through literature research,case review,questionnaire survey and qualitative interviews,9 dimensions are formed,including two dimensions of specific symptoms in the physiological field,non-specific symptoms,two dimensions of self-cognition and self-regulation in the psychological field,and disease cognition in the therapeutic field.Compliance,treatment evaluation,three dimensions,social support,social activities,two dimensions,a total of 71 items;(6)In the two rounds of inquiry,the recovery rate of the expert questionnaires is 100%,the expert authority coefficients are respectively 0.93 and 0.95 and the coefficient of variation(CV)fluctuates respectively between 0-0.32 and 0-0.15.Kendall's W value is 0.27 and 0.31.Delete 12 items,merge or modify 14 items,add 3 items and finally we got a total of 57 items of 9 dimensions in 4 fields,Conclusion:There are many problems in the physical,psychological,therapeutic and social aspects of patients with multiple myeloma,and each aspect affects each other,which seriously reduces the quality of life of patients.This study uses methods of literature research,case review,questionnaire survey,qualitative interview form the item pool of the MM-PRO scales,through two rounds of Delphi expert correspondence to modify the entries,and finally form the MM-PRO initial scale including 57 items of 9 dimensions in 4 fields,providing reference for subsequent research.