Efficacyr
Following 5 R-MPV cycles, 14 patients were in CR/CRu, 16 in PR, and 1 progressed (table 3). All patients in less than CR, in addition to 2 patients in CR/CRu with incomplete resolution of symptoms after 5 cycles, received 2 additional R-MPV cycles (N = 19). The objective response rate after R-MPV, defined as CR, CRu, or PR after 5 or 7 cycles in eligible patients with measurable disease (N = 31 evaluable) was 97% (95% confidence interval [CI], 83-100).
Thirty-one (97%) patients were deemed eligible for transplant based on response. A total of 26 (81%) patients eventually received HDC-ASCT. The other 5 patients did not undergo transplant because of refusal/consent withdrawal (N=2) or physician’s decision (N=3) and were removed from the study.
Among all patients (N = 32), the median PFS has not been reached, and the 1-year PFS estimate is 82% (95% CI, 62-92). There have been no events after 2 years, and the 2-year, 3-year, and 5-year PFS estimates are 79% (95% CI, 58-90).
The median overall survival (OS) has not been reached, and the 1-year OS is 88% (95% CI, 70-95). No deaths were observed beyond 2 years, with a 2-year, 3-year, and 5-year OS of 81% (95% CI, 63-91).
The median follow-up of survivors is 45 months (range: 27-86). Among the 26 transplanted patients, the 1-year PFS is 85% (95% CI, 64-94), and the 2-year, 3-year, and 5-year PFS estimates are 81% (95% CI, 60-92); the median PFS was not reached. The 1-year OS is 88% (95% CI, 68-96), and the 2-year, 3-year, and 5-year OS estimates are 81% (60% to 92%); the median OS was not reached.
The effects of age were analysed according to the MSK RPA class cutoff of 50. Patients age ≤50 years tended to achieve superior PFS (P = .050) and OS (P =0.06), with no observed progression or death of any cause.

© Blood 2015

© Blood 2015
Cognitive function and quality of life data
Neuropsychological testing showed that at baseline, there was evidence of impairment in several cognitive domains, characterizing baseline disease burden. Results of LMM analysis showed significant positive linear time components (months since baseline) for the Hopkins Verbal Learning Test - Revised (HVLT-R)–Delayed Recall and HVLT-R–Discrimination Index (P < 0.05), indicating continuous improvement in scores from baseline over time. Likewise, all of the other tests had significant positive linear components up to 12 to 18 months post-transplant. However, the rate of cognitive improvement slowed by 12 to 18 months post-transplant.r
Self-reported quality of life largely mirrored the improvement of cognitive function in the LMM analysis.The Functional Assessment of Cancer Therapy-Brain Cancer (FACT-BR) scores significantly improved from baseline, with slowed improvement by 12 to 18 months post-transplant; BDI scores significantly and linearly decreased over time.
Analysis of white matter abnormalities showed an improvement after R-MPV, with 81% of patients displaying scores 2 to 3 at baseline, compared with 19% after R-MPV (McNemar’s X² P = .002). Following transplant, there was an increase in white matter abnormalities, with 44% of patients with scores 2 to 3 (McNemar’s X² P = 0.046), which then remained stable over time. No scores above 3 were seen at any time.r