Pomalidomide for refractory and relapsed multiple myeloma: a retrospective cohort study of West of Scotland experience following Health Technology Assessment (HTA)

Kelly Baillie, Raymond Bernard Carragher, Christine Crearie, Jennifer Laskey, Marion Bennie, Grant McQuaker, Richard Soutar

Research output: Contribution to conferencePosterpeer-review


BackgroundMyeloma is predominantly a disease of the elderly, many of whom are excluded from clinical trials. In 2014, pomalidomide was accepted for use in combination with dexamethasone by the Scottish Medicines Consortium (SMC) for the treatment of relapsed and refractory multiple myeloma patients with at least two prior therapies. The trial reported a median progression free survival (PFS) and overall survival (OS) of 4 months and 12.7 months respectively. However, within their assessment SMC noted uncertainties relating to younger age and fitness of patients within the trial relative to expected eligible population in Scotland. It was also noted that the trial population may have been more heavily pre-treated (median 5 treatments).Aims & objectivesThe aim of this study was to use electronic record linkage (ERL) of healthcare data from patients treated across the four West of Scotland (WoS) Health Boards to describe characteristics and outcomes of pomalidomide patients relative to the trial population used to inform the SMC assessment. Objectives were: to describe OS and identify if PFS could be derived. Methods Patients treated with pomalidomide, between 2015 and 2017 within the WoS were identified using the Chemotherapy Electronic Prescribing and Administration System (CEPAS). This information was linked to data from the Prescribing Information System (PIS; community prescribing); the Scottish Cancer Registry (SMR06); Scottish Morbidity Records, inpatient episodes and outpatient appointments (SMR01 & SMR00); National Register of Scotland, death records (NRS) and the Scottish Care Information (SCI) store (laboratory test results) within the NHS GGC Safe Haven. Statistical analysis was performed using R®. OS and PFS was estimated using Kaplan-Meier method, and Cox’s proportional-hazards model was used to estimate unadjusted hazard ratios (HR) for the clinical variables.Results78 patients were identified; 65% were male and median age was 69 years (range 43-87). Performance status at baseline was 60% 0-1, 14% 2 and not available for 26%. Laboratory data was only readily available for patients treated in one health board (44%). Median number of prior treatments was 3 (Interquartile range 2-3). Data were censored on 30th November 2018 at which time; 76 patients had stopped and 20 remained alive (median follow up 12 months). The median duration of pomalidomide treatment was 3.9 months and median OS was 12.9 months. Poorer performance status and ≥3 prior treatments increased the death hazard (Unadjusted HR 4.15 (95% CI 1.74-9.93) and HR 1.72 (95% CI 1.01-2.92) respectively). Discussion/ConclusionWoS patients were older and had received fewer prior treatments, in line with the uncertainties highlighted within the SMC assessment. However, median OS was similar to that reported in the pivotal study. Description of PFS was hampered by inability to access all laboratory data for patients as SCI store is not a nationally held data set. Duration of therapy, often considered as a surrogate for PFS was nonetheless, similar to PFS reported in the trial.ERL is a feasible method to assess local outcomes when there are potential generalisability issues highlighted within the HTA assessment and may provide useful local context for patients, clinicians and funders.
Original languageEnglish
Publication statusPublished - 8 Oct 2021
EventBritish Oncology Pharmacy Association (BOPA) 24th Annual Symposium - Virtual
Duration: 8 Oct 202110 Oct 2021
Conference number: 24


ConferenceBritish Oncology Pharmacy Association (BOPA) 24th Annual Symposium


  • oncology
  • myeloma
  • pomalidomide
  • survival
  • electronic record usage
  • data linkage


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