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Neuro-Oncology 2004 6(3):253-258; doi:10.1215/S1152851703000607
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© 2004 by the Society forNeuro-Oncology

13-cis-Retinoic acid in the treatment of recurrent glioblastomamultiforme

Siew-Ju See, Victor A. Levin, W.-K. Alfred Yung, Kenneth R. Hess and Morris D. Groves1

Departments of Neuro-Oncology (S.-J.S., V.A.L.,W.-K.A.Y., M.D.G.) and Biostatistics (K.R.H.),University of Texas M.D. Anderson Cancer Center, Houston, TX 77030,USA

1 Address correspondence to Morris D. Groves, Department of Neuro-Oncology, Box431, University of Texas M.D. Anderson Cancer Center, 1515 Holcombe Blvd.,Houston, TX 77030, USA(mgroves{at}mdanderson.org).


  Abstract

Basic science and clinical investigations have demonstrated that13-cis-retinoic acid (cRA) has activity against malignant gliomas. Toassess its effectiveness in the setting of recurrent glioblastoma multiforme(GBM), we performed a retrospective analysis of the medical records andneuroimaging results of patients with recurrent GBM who were treated with cRA.The toxicity profile of cRA, response, and effect on progression-free survivalfrom initiation of treatment were end points of our analysis. Eighty-two of 85patients with a median age of 51 years received at least 1 full cycle of cRA.At the initiation of cRA treatment, the median Karnofsky performance score was80. All patients had failed conventional radiotherapy. Seven patients werechemonaïve, whereas 75 patients had received some form of chemotherapy.Radiographic partial responses, minor responses, and stable disease were seenin 4%, 8%, and 34% of patients, respectively. Two patients were notassessable. Progression-free survival and overall survival after initiation ofcRA were 10.0 and 24.6 weeks, respectively. Six-month progression-freesurvival was 19% for the entire group. Grade 3 or 4 toxicity developed in 14patients (16%), one of whom developed pancreatitis and died. The results ofthis study demonstrate only modest efficacy for cRA therapy in this cohort ofheavily pretreated patients with recurrent GBM. This data supports the use ofcRA in such patients, but its further evaluation in larger, prospective,controlled studies with or without other noncytotoxic and cytotoxic agents maybe warranted.

Received October 29, 2003; Accepted March 16, 2004


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