Efficacy and safety of MCNA in patients with nonmuscle invasive bladder cancer at high risk for recurrence and progression after failed treatment with bacillus Calmette-Guérin Journal Article


Authors: Morales, A.; Herr, H.; Steinberg, G.; Given, R.; Cohen, Z.; Amrhein, J.; Kamat, A. M.
Article Title: Efficacy and safety of MCNA in patients with nonmuscle invasive bladder cancer at high risk for recurrence and progression after failed treatment with bacillus Calmette-Guérin
Abstract: Purpose Patients with high risk recurrences after bacillus Calmette-Guérin failure have limited options. We performed an open label study to evaluate the efficacy and safety of intravesical MCNA in this setting. Materials and Methods Patients were treated intravesically with 8 mg MCNA weekly for 6 weeks followed by 3 weekly instillations at months 3, 6, 12, 18 and 24. Cystoscopy and cytology were performed every 3 months for 2 years with mandatory biopsy at 6 months and as clinically indicated thereafter. The primary efficacy end point was the disease-free survival rate at 1 year. Results A total of 129 patients were enrolled in study, including 91 with carcinoma in situ with or without papillary disease and 38 with papillary only tumors. Most patients had high risk disease. A total of 107 cases were bacillus Calmette-Guérin refractory and 2 or more prior bacillus Calmette-Guérin induction courses had been given in 68. Median followup in all patients was 34.7 months. The overall disease-free survival rate was 25.0% at 1 year and 19.0% at 2 years. In patients with papillary only tumors the disease-free survival rate was 35.1% and 32.2% at 1 and 2 years, respectively. The median disease-free duration in the 30 responders was 32.7 months. The progression-free survival rate was 87.3%, 79.8% and 77.7% at 1, 2 and 3 years, respectively, with a progression event in 28 patients. MCNA was well tolerated and few adverse events led to treatment discontinuation. Conclusions Intravesical MCNA achieved significant activity in patients at high risk with nonmuscle invasive bladder cancer in whom bacillus Calmette-Guérin treatment failed, especially those with papillary only tumors and those with bacillus Calmette-Guérin relapse. A durable response was seen, particularly in patients with a response at 1 year. MCNA offers an option for patients who are not candidates for or who refuse cystectomy. © 2015 American Urological Association Education and Research, Inc.
Keywords: cancer chemotherapy; cancer survival; controlled study; human tissue; cancer surgery; treatment failure; unclassified drug; major clinical study; overall survival; fatigue; cancer recurrence; cancer growth; cancer risk; drug efficacy; drug safety; drug withdrawal; cancer patient; disease free survival; recurrence risk; follow up; antineoplastic agent; bcg vaccine; cancer prevention; progression free survival; controlled clinical trial; tumor biopsy; drug screening; urinary bladder neoplasms; hematuria; multicenter study; carcinoma in situ; nucleic acids; cancer immunization; therapy delay; cystectomy; urinary tract infection; patient compliance; open study; urinary urgency; immunomodulating agent; cystoscopy; transurethral resection; mitomycin; drug treatment failure; drug tolerance; induction chemotherapy; dysuria; nucleic acid; bcg vaccination; urine cytology; bladder biopsy; maintenance chemotherapy; non muscle invasive bladder cancer; human; male; female; priority journal; article; mycobacterium phlei; mycobacterium phlei cell wall nucleic acid complex; drug instillation; pollakisuria
Journal Title: Journal of Urology
Volume: 193
Issue: 4
ISSN: 0022-5347
Publisher: Elsevier Science, Inc.  
Date Published: 2015-04-01
Start Page: 1135
End Page: 1143
Language: English
DOI: 10.1016/j.juro.2014.09.109
PROVIDER: scopus
PUBMED: 25286009
DOI/URL:
Notes: Export Date: 4 May 2015 -- Source: Scopus
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  1. Harry W Herr
    594 Herr