A National Registry of Patients Diagnosed with Multiple Myeloma in Taiwan.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 4955-4955
Author(s):  
Tzeon-Jye Chiou ◽  
Shang-Yi Huang ◽  
Cheng-Shyong Chang ◽  
Sheng-Fung Lin ◽  
Sung-Nan Pei ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Overall Response Rate ◽  
National Registry ◽  
Disease Stage ◽  
Line Treatment ◽  
Second Line ◽  
First Line ◽  
Overall Response ◽  
Best Response ◽  
First Line Treatment

Abstract Abstract 4955 Background The diagnosis and treatment pattern in actual practice for patients with multiple myeloma has not been described in Taiwan. This is the first national registry survey with the introduction of conventional and novel anti-myeloma therapies in Taiwan. Methods The “Multiple Myeloma Observational Study” is a national-based observational registry survey conducted by the Hematology Society of Taiwan. All patients newly diagnosed with multiple myeloma within one year are eligible for inclusion. Information on treatment, treatment response and survival was obtained by medical record review. We report 115 patients with multiple myeloma in Taiwan who were enrolled from January 2006 to December 2008. Results The characteristics of 115 patients were examined according to age, gender and disease stage. The average age was 65 years. The number of male patient was 72 (62.6%); female patient, 43 (37.4%). The proportions of disease stage were: stage I, 14 (12.2%); stage II, 26 (22.6%); stage III, 75 (65.2%). All patients had received anti-myeloma agents as their first line treatment since diagnosis. The most commonly used regimen was combination of melphalan, prednisolone and thalidomide in 27 patients (23.5%) and combination of thalidomide and dexamethasone in 27 patients (23.5%); followed by combination of melphalan and prednisolone, 24 (20.9%); combination of vincristine, anthracycline and dexamethasone (VAD), 15 (13%), thalidomide alone, 9 (7.8%); dexamethasone alone, 5 (4.3%); combination of VAD and thalidomide, 2 (1.7%); others, 3 (2.6%). The adjunct therapies along with first line treatment include zoledronic acid, 59 (51.3%); clodronate, 16 (13.9%); pamidronat, 8 (7.0%); and epoietin, 5 (4.3%). The outcomes of treatment were also recorded. The overall response rate for first line treatment was 59.1%. The best response of combination of VAD and thalidomide was 100%, followed by combination of thalidomide and dexamethasone, 66.7%; combination of melphalan and prednisolone, 66.7%; the combination of melphalan, prednisolone and thalidomide, 63%, combination of vincristine, anthracycline and dexamethasone, 60%; thalidomide alone, 22.2%; dexamethasone alone, 20%. A total of 31 patients had received anti-myeloma agents as the second line treatment. The most commonly used treatment was thalidomide alone, received by 7 patients (22.6%); followed by combination of thalidomide and dexamethasone, 5 (16.1%); combination of melphalan, prednisolone and thalidomide, 5 (16.1%); VAD, 5 (16.1%); combination of melphalan and prednilolone, 3 (9.7%); dexamethasone alone, 3 (9.7%) and other regimen, 3 (9.7%). The overall response rate for second line treatment was 41.9%. The best response of the combination of melphalan, prednisolone and thalidomide, 66.7%, followed by combination of vincristine, anthracycline and dexamethasone, 60%; thalidomide alone 57.1%; the combination of thalidomide and dexamethasone, 40%. The median survival duration was still not achieved yet. Conclusions The results of this national registry show that the choice of treatments in multiple myeloma covers a wide range of therapeutic modality. The overall response of first line treatment and second line treatment was comparable to the result of other registry studies. Disclosures Chiou: Janssen-Cilag Taiwan: Honoraria, Research Funding. Lin:Janssen-Cilag Taiwan: Honoraria. Chang:Janssen-Cilag Taiwan: Honoraria. Hsiao:Janssen-Cilag Taiwan: Honoraria.

Indirect Comparison of the Efficacy of Melphalan-Prednisone-Bortezomib Relative to Melphalan-Prednisone-Thalidomide and Melphalan-Prednisone for the First Line Treatment of Multiple Myeloma

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 2367-2367
Author(s):  
Yating Yeh ◽  
James Chambers ◽  
Sabine Gaugris ◽  
Jeroen Jansen
Keyword(s):  
Multiple Myeloma ◽  
Meta Analysis ◽  
Indirect Comparison ◽  
Complete Response ◽  
Similar Efficacy ◽  
Line Treatment ◽  
Relative Efficacy ◽  
First Line ◽  
Overall Response ◽  
First Line Treatment

Abstract Melphalan-prednisone (MP) combination has been considered a standard of care for front line treatment of multiple myeloma in patients non eligible for transplant. Melphalan-prednisone-bortezomib (MPV) combination has been approved in the United States in patients non eligible for high-dose chemotherapy (HD-C) and has recently received a positive opinion from the CHMP in Europe. Melphalan-prednisone-thalidomide (MPT) was approved in Europe in patients >65 or not eligible for HD-C. There is no head-to-head trial directly comparing MPV to MPT. The objective of the current study was to compare the efficacy of MPV to MP and MPT as first line treatment of multiple myeloma in patients non eligible for transplant. Six randomized placebo controlled trials investigating the efficacy of MPT (5) and MPV (1) relative to MP were identified with a systematic literature review. The endpoints of interest were overall survival (OS), progression free survival (PFS) and overall and complete response. Relative efficacy estimates of MPT versus MP as obtained from the MPT-MP trials were combined with meta-analysis techniques and simultaneously indirectly compared with the relative efficacy of MPV versus MP from the MPV-MP trial (VISTA). This adjusted indirect comparison was performed with Bayesian fixed and random effects models. As compared to frequentist approach, Bayesian meta-analysis offers a more informative summary of the likely value of efficacy after observing the data and allows for direct probabilistic inferences. Of the three interventions compared, there was an 81% probability that MPV was the most efficacious intervention in terms of overall response and a >99% probability in terms of complete response. With MPV a patient was two times more likely to show a complete response than with MPT (Relative Risk=2.15; 95%Credible Interval (CrI): 0.99–4.45). Both MPV and MPT showed greater OS than MP (HR=0.61; 95%CrI: 0.42–0.88 and HR=0.61; 95%CrI: 0.47–0.78 respectively); the indirect comparison showed similar efficacy in terms of OS between MPV and MPT (MPV vs MPT: Hazard Ratio=1.00; 95%CrI 0.64–1.57). Both MPV and MPT also displayed greater PFS than MP (MPV versus MP: HR=0.61; 95%CrI 0.49–0.76 and MPT versus MP HR=0.51; 95%CrI 0.41–0.63 respectively) and showed similar efficacy (MPV vs MPT: HR=1.19; 95%CrI: 0.87–1.63). In this study, both MPV and MPT are more efficacious than MP in terms of response, PFS and OS. MPV is expected to result in a greater complete and overall response than MPT. No difference in OS or PFS was displayed. Further analyses will need to be undertaken once evidence base data is more mature.

Coagulo-fibrinolytic activity as a predictor of efficacy in bevacizumab-combined chemotherapy in metastatic colorectal cancer patients.

2011 ◽  
Vol 29 (4_suppl) ◽  
pp. 533-533
Author(s):  
M. Suenaga ◽  
S. Matsusaka ◽  
T. Watanabe ◽  
K. Takagi ◽  
Y. Kuboki ◽  
...  
Keyword(s):  
Colorectal Cancer ◽  
Metastatic Colorectal Cancer ◽  
Fibrinolytic Activity ◽  
Line Treatment ◽  
Second Line ◽  
Predictors Of Outcome ◽  
First Line ◽  
Overall Response ◽  
D Dimer ◽  
First Line Treatment

533 Background: The combination of bevacizumab (BV) and chemotherapy in the first-line and second-line treatment of metastatic colorectal cancer (mCRC) has been shown to improve survival. Bevacizumab is a recombinant, humanized monoclonal antibody against vascular endothelial growth factor. However, the relationship between coagulo-fibrinolytic activity factors and treatment efficacy remains to be clarified. The aim of this study was to determine potential coagulo-fibrinolytic activity markers impacting survival. Methods: Among 119 consecutive patients included in the study, 85 received first-line FOLFOX4 plus BV 5 mg/kg and 34 received second-line FOLFIRI plus BV 5 mg/kg until progression of disease or unmanageable toxicity occurred. Coagulo-fibrinolytic activity factors, including D-dimer, thrombin antithrombin complex (TAT) and carbohydrate antigen 125 (CA125) encoded by the MUC16 mucin gene were evaluated as candidate predictors of outcome. Results: In first-line treatment, overall response, median progression-free survival (PFS) and two-year survival rate were 61.9%, 518 days and 67.3%, respectively. In second-line treatment, overall response, median PFS and median overall survival (OS) were 23.5%, 248 days and 651 days, respectively. The outcomes of the univariate analysis were as follows: normal D-dimer and CA125 levels at baseline were associated with better PFS and OS in first-line treatment; normal TAT and CA125 levels at baseline were associated with better PFS and OS in second-line treatment. According to the results of the multivariate analysis, normal D-dimer level was associated with longer PFS in first-line treatment, and only CA125 level at baseline was an independent predictor of both PFS and OS in second-line treatment. Conclusions: The results suggest that coagulo-fibrinolytic activity factors such as TAT, D-dimer or CA125 may be useful predictors of outcome in mCRC patients receiving BV in combination with chemotherapy. No significant financial relationships to disclose.

scholarly journals Real-World Effectiveness and Safety of Eltrombopag in Patients with Aplastic Anemia: A Chinese Nationwide Survey

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 5020-5020
Author(s):  
Wenrui Yang ◽  
Bing Han ◽  
Hong Chang ◽  
Bingyi Wu ◽  
Fankai Meng ◽  
...  
Keyword(s):  
Aplastic Anemia ◽  
Real World ◽  
Overall Response Rate ◽  
Response Rate ◽  
Line Treatment ◽  
First Line ◽  
Overall Response ◽  
Treatment Naïve ◽  
First Line Treatment

Immunosuppressive therapy (IST) based on antithymoglobin (ATG) and cyclosporin (CsA) is the first-line treatment for severe aplastic anemia (SAA) patients who are unfit for transplantation,and the overall response rate (ORR) is about 70%.The utility of eltrombopag (EPAG),a TPO receptor agonist, achieved robust hematologic response in refractory and treatment-naïve SAA patients in clinical trials and some other studies. However, only a few data came from Asia countries where higher incidence of AA has been reported. A retrospective study on the use of EPAG in AA was conducted in mainland China. Aplastic anemia (transfusion dependent non-severe, severe, and very severe) patients who started eltrombopag before Feb 2019 and continued for at least 3 months either as first-line treatment or as rescue treatment, were enrolled. The maximum daily dosage of EPAG used continuously for at least 2 weeks is defined as the stable dosage. Response criteria were referred to that used in previous reports (Townsley DM, NEJM 2017; BCSH, BJH 2016). Fifty-six patients from eleven centers were enrolled in this study, including 26 males and 30 females at the median age of 39 (7-80) years. All patients were transfusion-dependent by the time of EPAG administration, and there were 14 VSAA, 24 SAA and 18 transfusion dependent non-severe aplastic anemia (TD-NSAA). Nineteen treatment-naïve patients received EPAG and IST (ATG+CsA, n=10; CsA/CsA+androgen, n=9) as first-line treatment. Thirty-seven patients were refractory to IST. Eltrombopag was administered at a median dose of 75 (25-150) mg per day for 7 (3-31) month. The median follow-up time was 9 (3-40) months. The overall response rate in patients receiving EPAG as first-line therapy was 78.9% (15/19), and most patients achieved complete response (CR) (10/15). Among the 10 patients receiving ATG+CsA, 6 patients achieved hematologic response (HR) at 3 months post-treatment, including 3 CR. Six patients were diagnosed as VSAA and three achieved HR. For the 9 patients treated with CsA/CsA+androgen, 8 achieved HR (88.9%) and 4 were CR (44.4%) at 3 months. By last follow-up, the cumulative HR rate was 70% in ATG+CsA group and 89% in CsA/CsA+ androgen group. Among the 14 responders, 11 patients receiving EPAG at a stable dosage ≤75mg/d and achieved HR at 3 months. The overall response rate in IST-refractory patients was 46% (17/37), with trilineage response in 27% patients at 3 months. For the 18 ATG+CsA refractory SAA patients,trilineage HR occurred in 4 patients (22.2%, 4/18), bi-lineage HR in one patient and single lineage HR in one patient. Thus, the total HR was 33.3% (6/18) at 3 months and increased to 44% (8/18) by last follow-up. Among the 19 CsA/CsA+ androgen refractory patients, 6 (31.5%, 6/19) achieved trilineage HR, one achieved bi-lineage HR and 4 achieved single lineage response. Total HR rate was 57.9% (11/19) at 3 months after EPAG initiation and 68% (13/19) by last follow-up, including 9 patients with trilineage HR. Among 17 responders, 13 received a stable EPAG dose of≤75mg/d. Most patients tolerated EPAG well. Adverse events occurred in 29 patients (52%) and most were mild to moderate, including gastrointestinal symptom (n=15, e.g. abdominal pain, nausea), impaired liver function (n=5), skin changes (n=7, e.g. skin pruritus and rash) and musculoskeletal pain (n=6), and venous thrombus (n=2). Eltrombopag dosage was reduced in 2 patients due to severe digestive symptoms at 100 mg/d and discontinued in one patient who suffered from upper limb venous thrombus. In conclusion, EPAG is effective and safe in treating Chinese AA patients at a daily dose of 75mg and less. The real-world result of EPAG in Chinese patients is similar to those reported in Western countries. Disclosures No relevant conflicts of interest to declare.

Bevacizumab in Combination With Chemotherapy As First-Line Therapy in Advanced Gastric Cancer: A Randomized, Double-Blind, Placebo-Controlled Phase III Study

2011 ◽  
Vol 29 (30) ◽  
pp. 3968-3976 ◽  
Author(s):  
Atsushi Ohtsu ◽  
Manish A. Shah ◽  
Eric Van Cutsem ◽  
Sun Young Rha ◽  
Akira Sawaki ◽  
...  
Keyword(s):  
Gastric Cancer ◽  
Advanced Gastric Cancer ◽  
Overall Response Rate ◽  
Response Rate ◽  
Progression Free Survival ◽  
Line Treatment ◽  
First Line ◽  
Free Survival ◽  
Overall Response ◽  
First Line Treatment

Purpose The Avastin in Gastric Cancer (AVAGAST) trial was a multinational, randomized, placebo-controlled trial designed to evaluate the efficacy of adding bevacizumab to capecitabine-cisplatin in the first-line treatment of advanced gastric cancer. Patients and Methods Patients received bevacizumab 7.5 mg/kg or placebo followed by cisplatin 80 mg/m2 on day 1 plus capecitabine 1,000 mg/m2 twice daily for 14 days every 3 weeks. Fluorouracil was permitted in patients unable to take oral medications. Cisplatin was given for six cycles; capecitabine and bevacizumab were administered until disease progression or unacceptable toxicity. The primary end point was overall survival (OS). Log-rank test was used to test the OS difference. Results In all, 774 patients were enrolled; 387 were assigned to each treatment group (intention-to-treat population), and 517 deaths were observed. Median OS was 12.1 months with bevacizumab plus fluoropyrimidine-cisplatin and 10.1 months with placebo plus fluoropyrimidine-cisplatin (hazard ratio 0.87; 95% CI, 0.73 to 1.03; P = .1002). Both median progression-free survival (6.7 v 5.3 months; hazard ratio, 0.80; 95% CI, 0.68 to 0.93; P = .0037) and overall response rate (46.0% v 37.4%; P = .0315) were significantly improved with bevacizumab versus placebo. Preplanned subgroup analyses revealed regional differences in efficacy outcomes. The most common grade 3 to 5 adverse events were neutropenia (35%, bevacizumab plus fluoropyrimidine-cisplatin; 37%, placebo plus fluoropyrimidine-cisplatin), anemia (10% v 14%), and decreased appetite (8% v 11%). No new bevacizumab-related safety signals were identified. Conclusion Although AVAGAST did not reach its primary objective, adding bevacizumab to chemotherapy was associated with significant increases in progression-free survival and overall response rate in the first-line treatment of advanced gastric cancer.

scholarly journals Physicians' Behavior, Diagnostics and Treatment Patterns in Multiple Myeloma Management in Belarus: Nation-Wide Survey

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 5853-5853
Author(s):  
Ihar Iskrou ◽  
Anatoly Uss ◽  
Sergey Golubev ◽  
Vitali Papok
Keyword(s):  
Multiple Myeloma ◽  
Clinical Trials ◽  
Board Of Directors ◽  
Plasma Cells ◽  
Treatment Patterns ◽  
Line Treatment ◽  
Second Line ◽  
First Line ◽  
Advisory Committees ◽  
First Line Treatment

Abstract Background: Although multiple myeloma (MM) remains an incurable disease, its management progressed during the last decade owing to novelties in diagnostics and new therapeutic options. There is a general belief in heterogeneity of the novel technologies penetration among countries and regions, such differences should be studied. Information on physicians' knowledge, preferences and satisfaction is limited worldwide and may provide important insights for explanation of differences in clinical decision making in routine practice. Moreover, data on typical diagnostic and treatment patterns in real-world clinical setting are particular scarce in the Eastern European and Eurasian region. Methods: A cross-sectional national survey of physicians treating MM in Belarus was performed from October 2017 till January 2018. Among 51 hematologists registered in the country 43 physicians involved in MM management in real clinical settings were approached. Printed forms of 21-item questionnaire containing multiple choice questions were used. We anonymously collected physicians' opinions on typical diagnostics and treatment patterns as well as their clinical reasoning, preferences and satisfaction. We assessed whether practice place and type, practical experience (length of service and average number of MM patients seen per year) and attitude to participation in clinical trials influence answers. Univariate analysis was conducted with Fisher's exact test. Results: All approached physicians completed the survey. Among respondents 17 (40%) belonged to republican specialized centers, 37 (86%) were hospital-based physicians, 23 (53%) had more than 10 years of service, 28 (65%) seen more than 20 MM patients per year. 10 (23%) declared their experience in clinical trials and 20 (46%) had no experience but expressed readiness to be involved in. The clinical uptake of revised ISS for MM was 33%, among adopters physicians with more than 10 years in practice and who sees more than 20 patients per year dominated. The proportions of ISS users which believed that median survival for low-risk, standard-risk and high-risk MM patients to be > 12 months were 100%, 100% and 36%, respectively. For primary MM diagnosis 40% of respondents used MRI and 49% - CT-imaging. Physicians used the next criteria for treatment response : < 5% plasma cells (PCs) in bone marrow (88%), Ig level normalization (74%), absence of clonal PCs in BM (60%), and absence of new lesions (37%). The possibility to perform autologous stem-cells transplantation (ASCT) was revealed as a key factor for first-line treatment choice. Various bortezomib-based regimens were predominant treatment options for first-line treatment of patients eligible for ASCT. Melphalan-containing regimens were more widely spread as first-line treatment of ASCT-ineligible patients. The majority of respondents (52%) practiced first-line treatment of more than 4 months of duration, while 41% of clinicians used second-line therapy of short duration (less than 6 months). In the relapse setting after ASCT the most common regimens were still bortezomib-based as well as schemes with bendamustine. In the second-line setting in patients who did not receive ASCT monotherapy was more commonly reported. In absence of high dose dexamethasone for oral use Belarusian physicians preferred treatment schemes with combination of drugs for IV and per os routes of administration. The predominant factors of drug choice were efficacy (91%) and cost (97%). The respondents reported satisfaction with current situation in diagnostics and treatment in 74% and 65% of cases, respectively. The factors influencing readiness for disease management change were clinical experience, hospital-based practice position and positive attitude to/participation in clinical trials. Conclusions: The study covers the gaps of information about real-world MM management in Belarus. The Belarusian physicians are aware about the modern place of ASCT in MM. Targeted education in specific aspects of MM management (disease biology understanding, clinical guidelines updates, risk evaluation and stratification) may result in wider adoption of innovative diagnostic approaches and treatment technologies. MM management should be further concentrated in large specialized clinical centers for plasma cells disorders. The survey results make possible and warranted further intercountry comparisons. Disclosures Iskrou: Takeda Belarus: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Pfizer Belarus: Consultancy, Honoraria, Speakers Bureau; Novartis Belarus: Consultancy, Honoraria, Speakers Bureau; Nativita Belarus: Consultancy, Honoraria, Speakers Bureau; Octapharma Belarus: Consultancy, Honoraria, Speakers Bureau. Uss:Roche Belarus: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Takeda Belarus: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau. Golubev:Medical department of Takeda Belarus: Employment. Papok:Medical department of Takeda Belarus: Employment.

scholarly journals Bortezomib-based therapy in non-transplant multiple myeloma patients: a retrospective cohort study from the FABIO project

2021 ◽  
Vol 12 ◽  
pp. 204062072199648
Author(s):  
Matteo Franchi ◽  
Claudia Vener ◽  
Donatella Garau ◽  
Ursula Kirchmayer ◽  
Mirko Di Martino ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Cohort Study ◽  
Cost Effectiveness ◽  
Line Treatment ◽  
First Line ◽  
Conventional Chemotherapy ◽  
Risk Of Death ◽  
Model Average ◽  
Italian Regions ◽  
First Line Treatment

Introduction: Randomized clinical trials showed that bortezomib, in addition to conventional chemotherapy, improves survival and disease progression in multiple myeloma (MM) patients not eligible for stem cell transplantation. The aim of this retrospective population-based cohort study is the evaluation of both clinical and economic profile of bortezomib-based versus conventional chemotherapy in daily clinical practice. Methods: Healthcare utilization databases of six Italian regions were used to identify adult patients with non-transplant MM, who started a first-line therapy with bortezomib-based or conventional chemotherapy. Patients were matched by propensity score and were followed from treatment start until death, lost to follow-up or study end-point. Overall survival (OS) and restricted mean survival time (RMST) were estimated using the Kaplan–Meier method. Association between first-line treatment and risk of death was estimated by a conditional Cox proportional regression model. Average mean cumulative costs were estimated and compared between groups. Results: In the period 2010–2016, 3509 non-transplant MM patients met the inclusion criteria, of which 1157 treated with bortezomib-based therapy were matched to 1826 treated with conventional chemotherapy. Median OS and RMST were 33.9 and 27.9 months, and 42.9 and 38.4 months, respectively, in the two treatment arms. Overall, these values corresponded to a HR of death of 0.79 (95% CI 0.71–0.89) over a time horizon of 84 months. Average cumulative cost were 83,839 € and 54,499 €, respectively, corresponding to an incremental cost-effectiveness ratio of 54,333 € per year of life gained, a cost coherent with the willingness-to-pay thresholds frequently adopted from Western countries. Conclusions: These data suggested that, in a large cohort of non-transplant MM patients treated outside the experimental setting, first-line treatment with bortezomib-based therapy was associated with a favourable effectiveness and cost-effectiveness profile.

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