The determination of the number of patients required in a preliminary and a follow-up trial of a new chemotherapeutic agent

Gehan, Edmund A.

doi:10.1016/0021-9681(61)90060-1

Cited by 643 publications

(262 citation statements)

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“…The Gehan 2-stage design was used for estimating the response rate. 24 The sample size calculation was based on the double requirement of being able to stop the study early if the response rate was lower than 20% and of estimating the response rate with a standard error of <0.1, with an alpha of 0.05 and power of 95%. After the first stage comprising 14 patients had been completed, safety and efficacy were assessed.…”

Section: Discussionmentioning

confidence: 99%

Activity and safety of dose‐adjusted infusional cyclophosphamide, doxorubicin, vincristine, and prednisone chemotherapy with rituximab in very elderly patients with poor‐prognostic untreated diffuse large B‐cell non‐Hodgkin lymphoma

Musolino

Boggiani

Panebianco

et al. 2010

Cancer

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BACKGROUND: This study was designed to assess the activity and safety of dose-adjusted infusional cyclophosphamide, doxorubicin, vincristine, and prednisone chemotherapy with rituximab (DA-POCH-R) in elderly patients with poor-prognostic untreated diffuse large B-cell non-Hodgkin lymphoma (DLBCL). METHODS: From April 2006 to November 2009, 23 patients, aged !70 years, with an age-adjusted International Prognostic Index (IPI) of 2 or 3, were enrolled. Only patients with left ventricular ejection fraction (LVEF) !50% were allowed. The DA-POCH-R regimen was administered every 3 weeks for a minimum of 6 and a maximum of 8 cycles. RESULTS: Median patient age was 77 years (range, 70-90 years); 83% of patients had Ann Arbor stage III to IV disease. Median LVEF at baseline was 62%. Four (17%) patients had a history of abnormal cardiovascular conditions. Twenty-one (91%) patients were evaluable for response. The overall response rate was 90%, with a complete response rate of 57%. The 3-year overall survival and event-free survival rates were 56% and 54%, respectively. Neutropenia (48%) was the most frequent grade 3 to 4 adverse event (AE); no grade 3 to 4 cardiac AEs were observed. CONCLUSIONS: DA-POCH-R was an active and safe combination therapy for patients aged !70 years with poor-prognostic untreated DLBCL. This regimen was a reasonable alternative for elderly patients who were not considered to tolerate standard R-CHOP treatment. Cancer 2011;117:964-73.

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Section: Discussionmentioning

confidence: 99%

Activity and safety of dose‐adjusted infusional cyclophosphamide, doxorubicin, vincristine, and prednisone chemotherapy with rituximab in very elderly patients with poor‐prognostic untreated diffuse large B‐cell non‐Hodgkin lymphoma

Musolino

Boggiani

Panebianco

et al. 2010

Cancer

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“…Using standard statistical methods (Gehan, 1961), we used a 2-stage design in this study. If no CR or PR was noted in the first 14 patients, a response rate of 420% could be excluded with 95% confidence and accrual was stopped.…”

Section: Statistical Analysesmentioning

confidence: 99%

Weekly gemcitabine plus Epirubicin as effective chemotherapy for advanced pancreatic cancer: a multicenter phase II study

et al. 2002

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The current role of chemotherapy in pancreatic carcinoma is limited, and progress in the treatment of this disease represents a significant challenge to medical oncology. The most promising drug under study is gemcitabine, a relatively new antimetabolite that represents an attractive candidate for combination chemotherapy because of its excellent side-effect profile and the absence of overlapping toxicities with other chemotherapeutic agents. Combined administration of gemcitabine and anthracyclines could result in the induction of DNA breaks that are not easily repaired by the cell's machinery, thus enhancing the apoptotic signals triggered by these lesions. Forty-four patients with locally advanced and/or metastatic pancreatic adenocarcinoma were enrolled in this multicenter study. Patients received Epirubicin 20 mg m 72 for 3 weeks followed by 1 week of rest (1 cycle) and gemcitabine 1000 mg m 72 after Epirubicin on the same day. All were assessable for toxicity and response, 11 patients responded to treatment with one complete response and 10 partial responses, for an overall response rate of 25%. Median survival was 10.9 months (range, 2 -26 months). Therapy was well tolerated, with a low incidence of haematologic grade 42 toxicity. A total of 12 of 27 (44.4%) eligible patients attained a clinical benefit response. Our findings suggest that the gemcitabine-epirubicin schedule is active and well tolerated in patients with advanced pancreatic cancer.

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“…The MTD was defined as the dose level below the level at which two of three patients experienced DLT, provided no more than two of the six experienced DLT at that level. For phase II of the study, a Gehan 2 stage design (Gehan, 1961) was used. To detect a response rate of 15% with a probability of a type I error of 5% and a type II error of 10% required the enrolment of 21 patients in stage 1.…”

Section: Study Design and Statistical Methodsmentioning

confidence: 99%

A Phase I/II study of lomustine and temozolomide in patients with cerebral metastases from malignant melanoma

et al. 2006

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Temozolomide is an alkylating agent with activity in the treatment of melanoma metastatic to the brain. Lomustine is a nitrosurea that crosses the blood brain barrier and there is evidence to suggest that temozolomide may reverse resistance to lomustine. A multicentre phase I/II study was conducted to assess the maximum-tolerated dose (MTD), safety and efficacy of the combination of temozolomide and lomustine in melanoma metastatic to the brain. Increasing doses of temozolomide and lomustine were administered in phase I of the study to determine the MTD. Patients were treated at the MTD in phase II of the study to six cycles, disease progression or unacceptable toxicity. Twenty-six patients were enrolled in the study. In phase I of the study, the MTD was defined as temozolomide 150 mg m À2 days 1 -5 every 28 days and lomustine 60 mg m -2 on day 5 every 56 days. Dose-limiting neutropaenia and thrombocytopaenia were observed at higher doses. Twenty patients were treated at this dose in phase II of the study. No responses to therapy were observed. Median survival from starting chemotherapy was 2 months. The combination of temozolomide and lomustine in patients with brain metastases from melanoma does not demonstrate activity. The further evaluation of this combination therefore is not warranted.

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The determination of the number of patients required in a preliminary and a follow-up trial of a new chemotherapeutic agent

Cited by 643 publications

References 1 publication

Activity and safety of dose‐adjusted infusional cyclophosphamide, doxorubicin, vincristine, and prednisone chemotherapy with rituximab in very elderly patients with poor‐prognostic untreated diffuse large B‐cell non‐Hodgkin lymphoma

Activity and safety of dose‐adjusted infusional cyclophosphamide, doxorubicin, vincristine, and prednisone chemotherapy with rituximab in very elderly patients with poor‐prognostic untreated diffuse large B‐cell non‐Hodgkin lymphoma

Weekly gemcitabine plus Epirubicin as effective chemotherapy for advanced pancreatic cancer: a multicenter phase II study

A Phase I/II study of lomustine and temozolomide in patients with cerebral metastases from malignant melanoma

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