Long-term follow-up of pediatric MS patients starting treatment with injectable first-line agents: A multicentre, Italian, retrospective, observational study

2018 ◽  
Vol 25 (3) ◽  
pp. 399-407 ◽  
Author(s):  
Damiano Baroncini ◽  
Mauro Zaffaroni ◽  
Lucia Moiola ◽  
Lorena Lorefice ◽  
Giuseppe Fenu ◽  
...  
Keyword(s):  
Multivariate Analysis ◽  
Treatment Phase ◽  
First Line ◽  
Disease Evolution ◽  
Long Term Follow Up ◽  
Edss Score ◽  
Pre Treatment ◽  
Few Data

Background: Few data are available on very long-term follow-up of pediatric multiple sclerosis (MS) patients treated with disease modifying treatments (DMTs). Objectives: To present a long-term follow-up of a cohort of Pediatric-MS patients starting injectable first-line agents. Methods: Data regarding treatments, annualized relapse rate (ARR), Expanded Disability Status Scale (EDSS) score, and serious adverse event were collected. Baseline characteristics were tested in multivariate analysis to identify predictors of disease evolution. Results: In total, 97 patients were followed for 12.5 ± 3.3 years. They started therapy at 13.9 ± 2.1 years, 88 with interferons and 9 with copaxone. During the whole follow-up, 82 patients changed therapy, switching to immunosuppressors/second-line treatment in 58% of cases. Compared to pre-treatment phase, the ARR was significantly reduced during the first treatment (from 3.2 ± 2.6 to 0.7 ± 1.5, p < 0.001), and it remained low during the whole follow-up (0.3 ± 0.2, p < 0.001). At last observation, 40% had disability worsening, but EDSS score remained <4 in 89%. One patient died at age of 23 years due to MS. One case of natalizumab-related progressive multifocal encephalopathy (PML) was recorded. Starting therapy before 12 years of age resulted in a better course of disease in multivariate analysis. Conclusion: Pediatric-MS patients benefited from interferons/copaxone, but the majority had to switch to more powerful drugs. Starting therapy before 12 years of age could lead to a more favorable outcome.

Long-term follow-up of patients treated with glatiramer acetate: a multicentre, multinational extension of the European/Canadian double-blind, placebo-controlled, MRI-monitored trial

2007 ◽  
Vol 13 (4) ◽  
pp. 502-508 ◽  
Author(s):  
M. Rovaris ◽  
G. Comi ◽  
MA Rocca ◽  
P. Valsasina ◽  
D. Ladkani ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Glatiramer Acetate ◽  
Brain Mri ◽  
Treatment Phase ◽  
Double Blind ◽  
Brain Volumes ◽  
Disease Evolution ◽  
Long Term Follow Up

Glatiramer acetate (GA) is effective in reducing clinical and magnetic resonance imaging (MRI) activity in relapsing-remitting multiple sclerosis (RRMS). Serial long-term MRI data are lacking for large cohorts of GA-treated patients. The European/Canadian GA study consisted of two consecutive phases, each lasting nine months. The first treatment phase was randomized, double-blind and placebo-controlled. The second was an open-label, active treatment phase with daily administration of 20 mg GA subcutaneously for all patients. For the long-term follow-up (LTFU), dual echo, pre- and postgadolinium T1-weighted brain MRI scans were obtained with the same acquisition scheme as for the original trial and a neurological assessment was performed. Lesion volumes, normalized brain volumes and percentage brain volume changes (PBVC) were measured. One hundred and forty-two (63.4%) of the 224 patients who completed the two phases of the European/Canadian study underwent the LTFU after a mean period of 5.8 years (range: 5.3-6.4); 73 were treated with GA from study initiation. MRI measures at LTFU did not significantly differ between patients originally assigned to placebo and those who were always treated with GA, but the proportion of patients who did not require walking aids at LTFU was lower in the latter group (P=0.034). PBVC between baseline and LTFU was significantly correlated with lesion load at study entry. An earlier initiation of GA treatment in patients with active RRMS might, at least partially, have a favourable impact on long-term disease evolution. Multiple Sclerosis 2007; 13: 502-508. http://msj.sagepub.com

90Yttrium-Ibritumomab-Tiuxetan As First-Line Therapy Or As Consolidation Treatment In Advanced Stage Follicular Non-Hodgkin Lymphoma: Long-Term Results After a Median Follow-Up Of 61 Months

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 5123-5123
Author(s):  
Katharina Troppan ◽  
Angelika Valentin ◽  
Werner Linkesch, MD
Keyword(s):  
Advanced Stage ◽  
First Line ◽  
Ibritumomab Tiuxetan ◽  
Consolidation Treatment ◽  
Non Hodgkin Lymphoma ◽  
First Line Therapy ◽  
Significant Difference ◽  
Long Term Follow Up

Abstract Purpose Radioimmunotherapy combines biologic and radiolytic mechanisms to target and destroy tumor cells. 90Y-Ibritumomab-Tiuxetan (90YIT) is a murine anti-CD20 monoclonal antibody engaged for radioimmunotherapeutic targeting of CD20+ lymphoma cells. We report on our long-term follow-up data of 90YIT as first-line or consolidation treatment in advanced stage follicular Non-Hodgkin lymphoma (FL). Patients & Methods Between March 2004 and October 2010, forty-seven patients with CD20+ FL grade 1 to 3a in stages II, III, or IV were treated with a single dose of 90Yttrium-Ibritumomab-Tiuxetan (90YIT) at our institution. The median age was 61 years (range 41-83; male 55%) and 77% (n=36) of patients were in an advanced stage of the disease (stage III/IV). 90YIT was administered on an outpatient basis on day 8 after pretreatment with Rituximab (250mg/m²) on day 1. A mean of 1122 MBq (range 680-240) 90YIT was administered. Fourteen patients received 90YIT as first-line therapy, twenty-seven patients were treated with 90YIT after a median of 2 pretreatment courses (range 1-5) as consolidation therapy in remission (15 patients in CR, 12 patients in PR), and six patients showed progressive disease (PD) at time of 90YIT treatment. Median follow-up was 61 months (range 0-111). Results After a median follow-up of 61 months (range 0-111 months), 32 patients are still alive, including 21 patients in CR since 90YIT treatment. There was no significant difference concerning PFS and OS between first-line treatment and consolidation treatment, but we found a significant difference, comparing these two groups versus PD (PFS 51 months vs. 48 months vs. 8 months, p<0.023; OS 59 months vs. 71 months vs. 10 months, p<0.002) (figure 1 & 2). Survival rates were 85% (first-line), 67% (consolidation) and 33% (PD), respectively. Patients who maintained a CR after 90YIT treatment, showed significantly longer OS compared to patients with relapse after 90YIT (71 months vs. 52 months, p<0.001). No significant difference in PFS and OS was seen, concerning sex, age, or clinical stage. No unexpected toxicities emerged during long-term follow-up. Conclusion 90YIT as first-line, as well as consolidation therapy after achieving at least PR, provides a cost-efficient, long progression-free and overall survival in advanced stage FL. No benefit is shown in patients with PD, where we don't recommend 90YIT treatment. Disclosures: No relevant conflicts of interest to declare.

Autologous Hematopoietic Stem Cell Transplantation in Multiple Myeloma for Long-Term Follow-up: Single Center Results

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 5836-5836
Author(s):  
Weiwei Sui ◽  
Dehui Zou ◽  
Gang An ◽  
Shuhui Deng ◽  
Yan Xu ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Autologous Transplantation ◽  
First Line ◽  
Hematopoietic Stem ◽  
Agent Based ◽  
Total Treatment ◽  
Long Term Follow Up ◽  
Baseline Characteristics

Abstract Objective: To evaluate the efficacy and long-term outcome of the total treatment of induction therapy, ASCT and consolidation and maintenance therapy. Methods: A retrospective analysis was made on in multiple myeloma patients in our center between April 1, 2003 and February 1, 2016. The 157 patietns received autologous hematopoietic stem cell transplantation and review the autologous transplantation of long-term follow-up results. Analysis of the effect of transplantation efficacy, the impact on survival remission of different transplantation depth, transplantation in first line or not, salvage transplantation, prognosis of different staging system and other factors. Results: The baseline characteristics of the patients were shown in table 1. Overall patient ASCT before total effective rate (ORR) was 93.6%, in which the complete remission (CR) ratio was 33.1%. After ASCT, the best treatment response rate of PR was 80.3%, and the rate of CR was 58.6%. 91.69 months of median follow-up, patients with an overall survival (OS) and progression free survival (PFS) respectively 91.69 and 50.76 months; in 2005 before the median OS and PFS 39.0m and 23.0m. In 2005 after respectively and 56.41m 120.90m, P = 0.000. The median OS and PFS in the first line transplantation group and salvage transplantation group were vs 54.21m 39.0m and vs 7.09m 119.0m (P value was 0). 136 cases of patients with R-ISS stage, I, II, III of the patients with the median survival time were 120.90m (n=46), 86.43m (n=69), 35.65m (n=21), there were significant differences between groups, p=0.000. Each period of PFS were 72.11m, 51.84m, 28.09m, I and II, III,, p=0.001 and p=0.03, while there was no significant difference between II and III, p=0.122. The received autologous transplantation as first-line and salvage treatment of patients with subgroup survival analysis, median OS of the R-ISS stage III patients and different 15.84m 35.65m, P = 0.031; two groups of patients the median PFS (phase I: 91.69m vs18.92m; II: vs 16.69m 53.42m; phase III: vs 5.91m 28.52m) have difference (P = 0.000). In the first-line transplantation group, transplantation is more than or equal to PR and did not get effective PR group between OS were significantly different; before transplantation achieved CR, PR but did not obtain Cr and did not get effective PR group between PFS were significantly different; after transplantation and achieved CR CR did not get the patients had a median PFS were 65.57m 48.13m, P = 0.039 and median OS no difference. Accept any kind of noval agent- based chemotherapy were significantly longer OS and PFS than traditional chemotherapy (P = 0.001, P = 0.004) .There was no obvious difference on median OS between based regimen (bortezomib group median OS: NR; thalidomide group:120.90m); PFS in thalidomide group (median PFS : NR vs 54.21m) significantly prolonged (P = 0.010). By comparing the baseline characteristics of the two groups, it was found that the PFS was significantly shorter in the bortezomib group with an extra medullary lesion. Multivariate analysis showed that only R-ISS and the depth of remission before transplantation had effect on OS (p=0.003) and PFS (p=0.036) respectively. Conclusion: The total treatment of novel agent-based chemotherapy and ASCT for transplantation-eligible multiple myeloma patients is effective, further improve the remission rate and remission depth, prolong PFS and OS, the overall median survival up to 120.9m. First line transplantation can significantly prolong the OS and PFS compared with salvage transplantation. R-ISS and pre-transplant remission depth are prognostic factors influencing survival of patients. The total treatment to thalidomide based without extramedullary perhaps makes patients get long-term survival. Disclosures No relevant conflicts of interest to declare.

Antitumor activity of MDV3100 in pre- and post-docetaxel advanced prostate cancer: Long-term follow-up of a phase I/II study.

2011 ◽  
Vol 29 (7_suppl) ◽  
pp. 134-134 ◽  
Author(s):  
C. S. Higano ◽  
T. M. Beer ◽  
M. Taplin ◽  
E. Efstathiou ◽  
A. Anand ◽  
...  
Keyword(s):  
Prostate Cancer ◽  
Median Time ◽  
Radiographic Progression ◽  
Long Term Follow Up ◽  
Psa Progression ◽  
Previously Treated ◽  
Pre Treatment ◽  
Anti Tumor Activity

134 Background: MDV3100 is a novel androgen receptor (AR) antagonist selected for potent AR activity and devoid of partial agonist effects. A preliminary report of the phase I/II study described anti-tumor activity and adverse events (Scher HI et al. Lancet. 2010;375:1437). This abstract provides long-term follow-up on time to PSA and radiographic progression in this trial. Methods: Patients (pts) with progressive castration resistant prostate cancer (CRPC) were enrolled in sequential cohorts of 3-6 pts at MDV3100 doses of 30, 60, 150, 240, 360, 480 and 600 mg/day. Once the tolerability of a dose was established, enrollment was expanded at doses ≥60 mg/day to include approximately 12 chemotherapy naïve (naïve) pts and 12 pts previously treated with docetaxel (post-chemo) per cohort. Results: 140 pts were enrolled of which 18 (13%) pts continue on active treatment (16 naive and 2 post-chemo). The median time on treatment is 51 weeks for naïve and 17 weeks for post-chemo groups. Median time on treatment for the 18 patients still on study is 131 weeks. The median time to PSA progression, defined per-protocol as a ≥25% increase in PSA from baseline, was not met for naïve and was 33 weeks for post-chemo groups. Median time to PSA progression by Prostate Cancer Clinical Trials Working Group 2 criteria was 41 weeks for naïve and 20 weeks for post-chemo groups. Median time to radiographic progression was 56 weeks for naive and 24 weeks for post-chemo groups. Circulating tumor cell counts available for 128 of 140 pts showed 91% (70/77) with favorable pre-treatment counts (<5 cells/7.5 mL blood) remaining favorable post-treatment, while 49% (25/51) converted from unfavorable pre-treatment to favorable post-treatment. Conclusions: MDV3100 demonstrates durable anti-tumor activity in pts with CRPC both before and after chemotherapy. Based on these promising results MDV3100 is currently being evaluated in two global phase III studies in pts with metastatic CRPC, the AFFIRM study in pts previously treated with docetaxel and the PREVAIL study in chemotherapy-naïve pts who have progressed on androgen deprivation therapy. [Table: see text]

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