Targeted therapy in gastrointestinal stromal tumor (GIST): A nationwide database analysis from Taiwan.

2014 ◽  
Vol 32 (3_suppl) ◽  
pp. 437-437
Author(s):  
I-Jen Chiang ◽  
Wen-Yi Shau ◽  
Wei-Tse Fang ◽  
Chi-Hui Fang ◽  
Yen-Yang Chen

437 Background: Gastrointestinal stromal tumor (GIST) is a visceral sarcoma that arises from the gastrointestinal tract. Currently, imatinib (first-line) and sunitinib (second-line) are two reimbursed targeted therapies for GIST in Taiwan. This study aimed to evaluate the real world data of targeted therapies in GIST treatment among Taiwanese population. Methods: We conducted a nationwide retrospective cohort study based on data from the National Health Insurance Research Database (NHIRD) between January 2005 and December 2010. NHIRD is a comprehensive database of administrative and claim data which covers over 99% of entire population in Taiwan. Patients were selected with ICD codes and targeted therapy utilization, imatinib (IMA) or sunitinib (SUN). Time to treatment discontinuation (DC) due to any reasons (such as death, intolerance and disease progression) was estimated using Kaplan-Meier analysis. Results: From 2005 to 2010, the incidence rate of GIST in Taiwan was between 20 and 30 cases per million. A total of 3,436 GIST patients were identified; the mean age was 63.2 years and 57 % were male. Most patients (94.7%) received standard dose IMA (…400mg/day). Among these patients, 136 patients (4.0%) required IMA dose escalation and 44 (1.3%) patients switched to SUN after IMA. The probability of the 1st-line treatment DC was 20%, 30.7%, 38.9%, 43.2%, and 47.1% for years 1 to 5, respectively. The probability of a change in treatment pattern (defined as either IMA dose escalation or switch to SUN) was 4% 6.8%, 9.6%, 10.5% and 11.7% for years 1 to 5. Lastly, the probability of the 2nd-line treatment DC was 20.5%, 33.7%, 41.1%, 44.8%, 51.7% for years 1 to 5, respectively. Conclusions: First-line IMA plus other treatment modalities may provide additional benefits to progression-free survival. Taiwanese GIST patients who failed first-line treatment still gained benefit from either IMA dose escalation or a switch to SUN. Additional analysis is required for the detailed comparison in different treatment patterns.

2012 ◽  
Vol 30 (15_suppl) ◽  
pp. e16573-e16573
Author(s):  
May Hagiwara ◽  
Rohit Borker ◽  
Gerry Oster

e16573 Background: To estimate costs associated with adverse events (AEs) in patients receiving targeted therapies indicated for first-line treatment of mRCC. Methods: A retrospective study using a large US healthcare claims database (PharMetrics) from 1/2000 to 12/2009 was conducted. Study subjects were aged ≥18 years, had mRCC, and received first-line treatment with targeted therapies. AEs of interest comprised abdominal pain, back pain, diarrhea, dyspnea, extremity pain, fatigue/asthenia, hand-foot syndrome, hypertension, lymphopenia, nausea/vomiting, neutropenia, and proteinuria. Patients receiving care for these AEs were identified using ICD-9-CM diagnosis/procedure codes on healthcare claims. Costs were examined over a 30-day period, beginning with date of first mention of each AE; non-evented patients were assigned a “shadow” index date for comparison purposes. We estimated total costs over 30 days following the index date for patients with and without AEs, on both unadjusted basis and following adjustment for differences in baseline characteristics using a generalized linear model (GLM). Direct costs of targeted therapy were not considered. Results: Among patients receiving targeted therapy for mRCC, 64% had healthcare encounters for one or more AEs. AEs occurring with frequency >20% included severe abdominal pain, back pain, fatigue/asthenia, and nausea/vomiting, respectively; 10-20% of patients had encounters for diarrhea, dyspnea, and extremity pain, respectively. Mean [SD] total costs of care during the 30-day, post-index period were substantially higher among patients with AEs ($12,177 [$19,621] vs $4070 [$8142] for those without AEs). Adjusting for differences in baseline characteristics, the estimated cost difference (95% CI) was $11,373 ($5286 - $21,419). Conclusions: Costs associated with AEs of first-line targeted therapies are substantial in patients with mRCC. Efforts to prevent and/or better manage these events may reduce healthcare costs.


2021 ◽  
Vol 4 ◽  
pp. 1-1
Author(s):  
Philippos Apolinario Costa ◽  
Caroline Kamal Abdelmessih Hana ◽  
Navin Chakravarthy Balaji ◽  
Anthony Frank Skryd ◽  
Brianna Nicole Valdes ◽  
...  

2012 ◽  
Vol 30 (15_suppl) ◽  
pp. e15049-e15049
Author(s):  
Antonin Levy ◽  
Jean Menard ◽  
Laurence Albiges ◽  
Mario Di Palma ◽  
Karim Fizazi ◽  
...  

e15049 Background: Sequential treatment is currently the standard of care in metastatic renal cell carcinoma (mRCC). However, very little is known on how many patients (pts) can receive second line or further, and on how to predict those pts. Methods: Data from all mRCC patients treated at the IGR from 2005 to 2009 with first-line targeted therapy (sunitinib (SU), sorafenib (SO), bevacizumab (B), temsirolimus or everolimus (pooled together as mTOR)) were analyzed. Only patients with subsequent follow up have been included in this analysis. Patients were defined as “non eligible” for second treatment if: they were (i) still on first line treatment, (ii) not showing progressive (durable stable disease or partial response or complete response), or (iii) if they refused a second line treatment. Results: 251 patients, median age 60 years, median follow-up 20.2 months were treated with targeted therapy with a median OS of 25.8 months. Median OS with SU (127), SO (60) or B (61) were respectively 26.3, 16.4 and 32.5 months respectively. Only 3 patients received an mTOR inhibitor as first line. According to the eligibility criteria, the percentage of patients who received a second line was 59% (n=61/103), 52% (n=30/58) and 79% (n=38/48) for Su, So and B, respectively. MSKCC classification (p = 0.02) and first line agent (p = 0.001) were significant predictive factor for receiving a second line of treatment. Overall, patients receiving B were in better general condition, with 77% of PS = 0 compared to SO (53%) and SU (48%) (p = 0.005). Among the 131 patients who received a second line, the median OS from the start of second line treatment was 20.8 months for a TKI (n=98; 75%) and 16.6 months for an mTOR (n=32; 42%) (p = 0.12). Furthermore, the percentage of patients who received a third line was 56% (27/48), 28% (7/25) and 65% (13/20) for SU, SO and B, respectively. Conclusions: The median OS in patients treated with targeted therapies for mRCC in The Institut Gustave Roussy exceeds 2 years. The use of second line varies from 52% to 79%. Further studies are needed to validate the MSKCC groups and first line therapy as predictive factor for 2nd line treatment.


2009 ◽  
Vol 27 (15_suppl) ◽  
pp. e15602-e15602
Author(s):  
J. A. Morgan ◽  
A. Guo ◽  
D. Williams ◽  
A. Guérin ◽  
D. Latremouille-Viau ◽  
...  

e15602 Background: Imatinib is accepted as the standard first-line therapy to treat gastrointestinal stromal tumor (GIST) in patients with unresectable or metastatic disease. In the case of disease progression, physicians may consider increasing imatinib dose or switching to another second-line agent, such as sunitinib. The aim of this study was to analyze the real-world long term treatment patterns of GIST patients. Methods: Two large claims databases from 01/1999 to 03/2008 were combined (MarketScan and Ingenix Impact) to extract patients diagnosed with GIST who initiated on imatinib ≥400 mg/day. Patients were followed from the first observed imatinib prescription to the end of data availability. Patients who dose increased during the study period were defined as dose escalators, while patients who later switched to sunitinib with or without dose escalation were defined as switchers. Kaplan Meier analyses were used to estimate the rate of treatment changes over time including imatinib discontinuation, defined as a lack of imatinib supply for ≥60 days, and switching back to initial treatment. Results: Among the 1,508 GIST patients who initiated on imatinib, 253 patients had a dose increase and 153 patients switched to sunitinib during the study period. 20.1% patients on imatinib discontinued by the end of the first year, and 43.0% discontinued by the end of the third year. Among the 153 switchers, 61 patients had a dose escalation prior to the switch, of which 82.0% reached 800 mg before switching. Among all the switchers, 20.3% switched back to imatinib within 6 months. Conclusions: These claims-based findings on the duration of first-line imatinib therapy of GIST patients supports data on imatinib efficacy in treating GIST from formal clinical research studies. Most of GIST patients who initiated imatinib continued on this therapy without undergoing dose increase or switch to sunitinib. Among switchers, most did not dose escalate before switching and many eventually switched back to imatinib. It appears that physicians tend to not always follow clinical practice guidelines developed based on expert consensus, with respect to decision rules for dose escalation, or for the appropriate time to change kinase inhibitor therapy. [Table: see text]


2021 ◽  
pp. 1-6
Author(s):  
Bita Geramizadeh ◽  
Alireza Shojazadeh

<b><i>Background:</i></b> Gastrointestinal stromal tumor (GIST) of the ampulla of Vater is a rare occurrence. To the best of our knowledge, there has been no published review on this rare tumor in the English literature so far. <b><i>Summary:</i></b> In this review, we will discuss all the reported details of the published cases, including demography, clinical presentation, imaging, gross pathology and histopathology, immunohistochemical findings, treatment modalities, and outcome of cases with the diagnosis GIST from the ampulla of Vater in the last 20 years. <b><i>Key Message:</i></b> Twenty-five cases of GIST in the ampulla of Vater have been reported in the last 20 years in the English literature. GIST in the ampulla of Vater are usually small tumors (&#x3c;5 cm) in middle-age patients. The majority of the patients present with lower GI bleeding and abdominal pain. Imaging findings are not characteristic, and most of the patients without biopsy and with no histologic diagnosis were operated with the primary impression of adenocarcinoma, neuroendocrine tumor, and GIST. Perioperative tissue biopsy has been accurate in &#x3c;70% of the cases. The majority of the reported cases of GISTs in the ampulla of Vater have been low risk with spindle-cell morphology, low mitotic figures, and minimal atypia; reactive for C-KIT and DOG-1; and nonreactive for SMA, desmin, and S100. In the majority of the cases, duodenectomy with or without Whipple’s operation has been performed, and most of the cases showed good prognosis.


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