Evaluation of natural history and health care resource utilisation of patients with monocloncal gammopathy of unknown significance and myeloma bone disease

Study type
Protocol
Date of Approval
Study reference ID
18_137
Lay Summary

Myeloma is a rare bone marrow cancer with 5,500 patients diagnosed annually in the UK. Treatment is usually not curative and patients often have bone complications including pain and fractures. Before patients get myeloma, they always have an abnormal protein in their blood called a paraprotein. This pre-cancerous stage is called monoclonal gammopathy of uncertain significance (MGUS). Paraproteins need a special laboratory test not routinely performed and some people with myeloma will have previous diagnosis of MGUS. We do not know why only some patients with MGUS develop myeloma. Identifying patients at high risk of complications is important to guide treatment choices. The aim of this study is to provide evidence to guide treatment decisions for MGUS and myeloma for doctors and patients

To ensure the CPRD clinical or medical information in the Clinical Practice Research Datalink codes are determining the correct patients, questionnaires will be sent to a sample of General Practitioners to answer questions about patients who are registered at their practices with a possible diagnosis of myeloma or MGUS. We will compare their records with patients of a similar age, gender, ethnicity and from the same practice to understand who gets complications and how they use healthcare services.

Technical Summary

Myeloma is a rare cancer of the bone marrow. While the clinical focus is on treating myeloma, bone complications are a major concern to patients. Myeloma is always preceded by a premalignant condition called monoclonal gammopathy of unknown significance (MGUS), diagnosed by measuring special proteins called paraproteins in the blood and urine. Having MGUS is also associated with an increased risk of bone complications1 as well as progression to myeloma.
The aim of this study is to provide evidence to inform the clinical decision-making and provision of health care services to reduce the burden of bone complications in MGUS and myeloma2.
We will use the Clinical Practice Research Datalink verification study where the GPs General Practitioners of potential patients are sent a brief anonymous questionnaire to validate the patients specific diagnoses. Regression methods, including generalised linear models, will be used to describe the other diagnoses/ procedures to understand the natural history, health care usage and costs for people with MGUS and myeloma and their determinants using CPRD and where available linkage to HES. We will compare these findings with up to 4 controls of the same age, gender, ethnicity and GP practice.

Health Outcomes to be Measured

Primary outcomes:
- Bone fracture
Secondary outcomes:
- Prevalence
- Natural history
- Complications
- Progression of MGUS to myeloma
- Health care use and costs

Collaborators

Kassim Javaid - Chief Investigator - University of Oxford
Kassim Javaid - Corresponding Applicant - University of Oxford
Antonella Delmestri - Collaborator - University of Oxford
Constantinos Koshiaris - Collaborator - University of Oxford
Daniel Prieto-Alhambra - Collaborator - University of Oxford
Rafael Pinedo-Villanueva - Collaborator - University of Oxford
Roshi Shrestha - Collaborator - University of Oxford
Samuel Hawley - Collaborator - University of Bristol

Former Collaborators

Mital Kinderkhedia - Collaborator - University of Oxford

Linkages

HES Accident and Emergency;HES Admitted Patient Care;HES Diagnostic Imaging Dataset;HES Outpatient;ONS Death Registration Data;Patient Level Index of Multiple Deprivation