Symptomatic Multiple Myeloma (MM) is a rare cancer of the plasma cells, a type of white blood cell that originate in the bone marrow. Protection against organ damage in MM patients can be achieved with early diagnosis, early initiation of treatment and early prevention of bone complications. Diagnosis is challenging as it has a range of non-specific symptoms including bone pain, fatigue and fractures, which can often mean the disease goes undiagnosed or is diagnosed at a later stage. General Practitioners (GP) play a significant role in referrals of newly diagnosed patients to specialists for diagnosis and treatment - MM is the cancer with the highest percentage of patients consulting their GP three or more times before referral. This project serves to estimate the frequency of presenting symptoms within the GP setting in newly diagnosed MM patients, helping to understand the conditions that may predispose MM.
Diagnosing symptomatic Multiple Myeloma (MM) is challenging, as it presents with a myriad of symptoms, with non-specific and multiple site symptoms such as bone pain, fatigue, fracture, etc. Given that GPs play a significant role in referrals of newly diagnosed multiple myeloma patients (NDMM), the primary care setting represents an excellent milieu to investigate the aetiology of diagnosis. This is the scientific research focus in this retrospective population-based cohort study in the United Kingdom (UK).
The main aim is to estimate the frequency of presenting initial symptoms of MM patients at their first point of contact with the health care system (within the routine primary care setting) in the UK. The primary objectives are to estimate the proportion of MM patients with a record of bone pain, CRAB criteria investigations (elevated Calcium, Renal Failure, Anaemia, Bone Lesions), and positive CRAB criteria in a cohort of NDMM patients within the primary care setting, and to describe the patients in terms of demographic and clinical characteristics. The secondary objective is to assess the reproducibility of the primary analysis results using two analytical approaches, namely Aetion Evidence Platform and SAS statistical software. Exploratory objectives will explore the primary endpoints in specific sub-cohorts, namely NDMM patients with a record of bone pain and/or skeletal-related events (SREs).
This study is descriptive in nature, no formal hypotheses will be tested. Summary statistics will be reported for continuous variables and will include estimates of the mean, with number of observations and standard deviation reported to indicate precision. For categorical variables, proportions will be calculated and numbers of patients in each group will be reported.
Primary: Record of bone pain; time from first-ever bone pain record to MM diagnosis; time from MM diagnosis to first-ever bone pain record; record of CRAB criteria investigations; record of at least 1 confirmatory test result for positive CRAB criteria; number of recorded test results
Secondary: Reproducibility of the primary analysis results using two analytical approaches
Exploratory: Record of bone pain among a subset of patients with a record of SRE(s)
Key variables
Bone pain; Patient demographics Age; Gender; Length of pre-index time available; Length of follow-up available;
Clinical characteristics - history of diagnosed comorbidities; history of pathological fractures; concomitant medication; CRAB criteria investigations and positive CRAB criteria - hypercalcaemia and confirmed hypercalcaemia; renal impairment and confirmed renal impairment; anemia and confirmed anaemia; imaging for investigating bone and confirmed bone/lytic lesion(s).
Anouchka Seesaghur - Chief Investigator - Amgen Ltd
Victoria Banks - Corresponding Applicant - Amgen Ltd
David Neasham - Collaborator - Amgen Ltd
Edwin Hoeben - Collaborator - Amgen Ltd
George Kafatos - Collaborator - Amgen Ltd
Joe Maskell - Collaborator - Amgen Ltd
Natalia Petruski-Ivleva - Collaborator - Aetion, Inc
Shannon Reynolds - Collaborator - Aetion, Inc