Improved outcome in patients following autologous stem cell transplantation for multiple myeloma in south eastern Norway 2001-2010: a retrospective, population based analysis.

BACKGROUND: With the advent of novel drugs improved overall survival in patients with multiple myeloma, including patients who received up-front autologous stem cell transplantation (ASCT), has been reported from several centers. Here we report on overall survival in a population-based cohort of patients receiving ASCT as first line treatment and in whom novel agents were an option for second and later lines of treatment. METHODS: Patients with multiple myeloma ≤ 65 years of age who were considered for ASCT from 01.01.2001-31.06.2005 (period 1) and from 01.07.2005 until 31.12.2009 (period 2) at Oslo University Hospital (OUH) were identified. Relevant data were collected from the patients' medical records. RESULTS: Altogether, 293/355 patients received ASCT. In all, median OS was 82.9 months in patients ≤ 60 years of age and 59.0 months in patients 61-65 years. For patients ≤ 60 years of age median OS increased from 70.6 months to 87.7 months (p = 0. 22) and median survival after start of second line therapy increased from 34.5 months to 46.5 months (p = 0.015) between the two periods. For patients 61-65 years of age median OS increased from 57.3 months to 61.2 months (p = 0. 87) and median survival after start of second line therapy was practically unchanged (32.6 months vs. 33.1 months (p = 0.97) between the periods. In patients ≤ 60 years of age salvage ASCT was used in 34% of the patients while in patients 61-65 years of age salvage ASCT was used in 7.3% of the patients. The use of salvage ASCT and novel drugs, as well as the number of treatment lines, were higher in patients ≤ 60 years of age and increased during the study period. CONCLUSION: In patients ≤ 60 years of age an increased median OS of 17 months between the two periods were noted, but the difference failed to reach statistical significance. However, a statistically significant difference in median survival of 12 months after start of second line therapy was found in this age group, which may be explained by a more active second line treatment. In patients 61-65 years only a slight increase of survival, not statistically significant, was noted between the periods.

Bone scintigraphy reduces the need for biopsy in suspected cardiac amyloidosis. / Skjelettscintigrafi reduserer behovet for biopsi ved mistanke om kardial amyloidose.

The gold standard to diagnose suspected cardiac amyloidosis is myocardial biopsy. In recent years, bone scintigraphy has partly replaced myocardial biopsy.

Estimated Financial Savings From Clinical Studies at Oslo Myeloma Center in the Period 2015-2021.

BACKGROUND: The number of clinical studies in the Western world has been declining the last decade. Clinical studies offer valuable opportunities for cancer patients to access new treatments and serve as arenas for learning and competence development for health care workers. In addition to this, clinical studies can significantly contribute to financial savings for the health care system through the provision of drugs. The extent of these savings have not been evaluated before. MATERIALS AND METHODS: We assessed the financial savings from drugs provided in clinical studies conducted at Oslo Myeloma Center between 2015 and 2021. Only drugs that had marketing license or another equivalent drug with marketing license were considered to estimate savings. RESULTS: A total of 314 patients across 24 different studies were given treatment with drugs possessing marketing license. Drugs approved for clinical use and reimbursed by the national health care system gave a financial saving of 20.3 million USD and was considered a direct saving. Drugs not approved for clinical use, but having equivalent approved alternatives yielded a financial saving of 4.7 million USD. CONCLUSION: Clinical studies not only offer new opportunities for patients and advancements in medical treatment and knowledge but also contribute significantly to financial saving for the health care system through reduced drug expenses.

[High-dose treatment of systemic AL-amyloidosis with autologous stem cell support]. / Høydosebehandling med autolog stamcellestøtte ved systemisk AL-amyloidose.

BACKGROUND: AL-amyloidosis is a serious disease with a short median survival without treatment. Treatment with high-dose melphalan with autologous stem cell support (HMAS) has a potential to increase survival, but is associated with toxicity and mortality. In this paper we report the Norwegian results retrospectively. MATERIAL AND METHODS: We used questionnaires and had personal contact with a local physician from each hospital with HMAS experience. Diagnosis and treatment were evaluated according to the guidelines at the time of treatment, and the results were compared to internationally published reports. RESULTS: Stem cell harvesting was attempted in 18 patients from 1997 to 2006. 15 of these received HMAS treatment. Treatment-related mortality was 20%, and 5 of 11 (45%) had an organ response. Median survival was not reached within the 55-month median observation time. The course of the disease was more complicated when known risk factors for HMAS treatment were present, such as reduced kidney function, advanced heart involvement, reduced performance status, and multiorgan disease. Three of 18 patients were not diagnosed according to relevant guidelines. In seven of 12 patients the response to treatment was not evaluated adequately with respect to haematology. INTERPRETATION: AL-amyloidosis is a difficult diagnosis and the condition is probably under-diagnosed in Norway. The results of HMAS treatment in Norway are comparable with those in published reports from centres abroad. The follow-up of patients should be improved.