One-Year Progression-Free Survival of Therapy-Naive Patients With Malignant Pheochromocytoma and Paraganglioma

Abstract Context: The natural history of malignant pheochromocytoma or paragangliomas (MPP) remain unknown. Objective: The primary aim of this study was to define progression-free survival at 1 year in therapy-naive patients with MPP. Secondary objectives were to characterize MPP and to look for prognostic parameters for progression at 1 year. Design and Setting: The files of MPP followed up between January 2001 and January 2011 in two French Endocrine Networks were retrospectively reviewed. Therapy-naive patients were enrolled. Main Outcome Measures: The main outcome was progression-free survival at 1 year in therapy-naive MPP patients according to Response Evaluation Criteria In Solid Tumors 1.1 criteria. Results: Ninety files (46 men, 44 women, mean age of 47.5 ± 15 years) were reviewed on site by one investigator. MPP characteristics were as follows: presence of an adrenal primary, a mitotic count exceeding 5 per high power field, hypertension, inherited disease, and presence of bone metastases in 50%, 22%, 60%, 49%, and 56% patients, respectively. Fifty-seven of the 90 patients with MPP (63%) were classified as therapy-naive. The median follow-up of these 57 patients was 2.4 years (range, 0.4–5.7). At 1 year, progression-free survival was 46% (CI 95: 33–59). Twenty-six of 30 (87%) patients with progression at 1 year had exhibited progressive disease at the first imaging workup performed after a median of 5.7 months. No prognostic parameter was identified. Conclusions: Half of the therapy-naive patients with MPP achieved stable disease at 1 year. In symptom-free patients with MPP, a wait-and-see antitumor policy seems appropriate as first line. Modality for a prospective follow-up is proposed.

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Bortezomib, Thalidomide, and Dexamethasone as Induction Therapy for Patients with Symptomatic Multiple Myeloma.

Blood ◽

10.1182/blood.v110.11.3605.3605 ◽

2007 ◽

Vol 110 (11) ◽

pp. 3605-3605 ◽

Cited By ~ 1

Author(s):

Jonathan L. Kaufman ◽

Charise Gleason ◽

Leonard Heffner ◽

Sagar Lonial

Keyword(s):

Induction Therapy ◽

Progressive Disease ◽

Response Rate ◽

Progression Free Survival ◽

High Response Rate ◽

High Dose ◽

High Dose Therapy ◽

Free Survival ◽

One Year

Abstract The optimal induction regimen for patients with symptomatic myeloma who are eligible for transplantation is currently unknown. While thalidomide and dexamethasone is an effective regimen, it only has a 60 to 65% response rate and few complete responses (CR). Bortezomib based inductions have demonstrated a high response rate and an improved CR as well. Recently the IFM reported the initial results of the randomized bortezomib plus dexamethasone versus VAD induction followed by transplant, which demonstrated that fewer patients treated with bortezomib required tandem transplants. Wang et al reported a high induction response rate with the combination of BTD for only 2 cycles given over a 28 day cycle. Here we report our experience with the combination of BTD as induction therapy. 38 patients with symptomatic myeloma were treated with BTD as induction therapy. Patients received standard dose and schedule bortezomib at 1.3 mg/m2 on days 1, 4, 8, and 11 with thalidomide at 100 mg/day, and 8 days of 40 mg dexamethasone every 21 days. The median age was 58 years (38–70) with 19 males. This was first line therapy for 29 patients, second line for 7 patients and 3rd line for 2. 12 patients had ISS stage 2 and 8 had ISS stage 3. The median β2M was 3.4 (1.66–41.89). Median creatinine was 1.1 (0.6–21.0). Nineteen patients had an IgG paraprotein, 6 an IgA, and 16 patients had light chain disease. The median number of cycles administered was 4 (2–8). Fifteen patients developed neuropathy of any grade. One patient developed grade 3 neuropathy. The overall response rate (CR, VGPR, plus PR) was 92%, with 58% of patients achieving a VGPR or better, and 21% of patients achieving an immunofixation negative CR. 1 patient had a minimal response and 2 patients had progressive disease (both patients presented with plasma cell leukemia). These two patients were treated with the combination of BTD with PACE chemotherapy. One of the two died from progressive disease and the other patient remains in complete remission after high dose therapy and autologous transplantation. 29 patients had consolidation therapy with high dose melphalan and autologous peripheral blood stem cell transplantation. Eight patients have collected stem cells without proceeding with immediate consolidation therapy. After a median follow up of 373 days, median progression free survival and overall survival have not been reached. One year overall survival is 97%. One year progression free survival is 87%. In conclusion, we report a very high response rate with a short course of bortezomib, thalidomide and dexamethasone with an acceptable toxicity profile. Follow up of patients in CR treated without high dose therapy and autologous transplant is in progress. Further studies of this active regimen are warranted.

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Radiation-associated versus sporadic osteosarcoma: A single-institution experience.

Journal of Clinical Oncology ◽

10.1200/jco.2017.35.15_suppl.11018 ◽

2017 ◽

Vol 35 (15_suppl) ◽

pp. 11018-11018

Author(s):

Brittany Siontis ◽

Emily Roberts ◽

Lili Zhao ◽

Jonathan B. McHugh ◽

Dawn Owen ◽

...

Keyword(s):

Rare Complication ◽

Progression Free Survival ◽

Primary Malignancy ◽

Free Survival ◽

Significant Difference ◽

Detectable Difference ◽

History Of ◽

Median Cumulative Dose ◽

Worse Prognosis

11018 Background: Osteosarcoma (osarc) can be a rare complication from radiation (rt) therapy. Radiation-associated osarc (RAO) is reported to have a worse prognosis than non rt-associated osarc with limited objective data comparing the two. We conducted a retrospective study comparing demographics, therapy and outcomes of sporadic osarc (SO) to RAO. This study was confined to adults. Methods: We identified patients (pts) > age 18 years (yr) with osarc treated at our institution between 1990 and 2016 using an institutional database. We categorized tumors as SO or RAO based on history of prior rt within field of osarc. We extracted data on demographics, treatment, and primary malignancy characteristics. Results: We identified 159 pts with osarc, 28 were RAO tumors. Results are in Table 1. Median follow-up was 2.8 yr (0.1-19.6 yr). For RAO, median time from rt to diagnosis was 11.5 yr (1.5-28 yr) with a median cumulative dose of 60 Gy (44-75.8 Gy). Median progression free survival (PFS) and overall survival (OS) were not significantly different in pts presenting with metastatic osarc; PFS 10.3 mo vs 4.8 mo (p=0.45) and OS 15.6 mo vs 6.1 mo (p=0.96) in SO vs RAO pts, respectively. For pts with localized osarc, median relapse-free survival (RFS) and OS were significantly different, not reached vs 12.2 mo (p<0.001) and not reached vs 27.6 mo (p=0.001) in SO vs RAO, respectively. Conclusions: In our series, there was a significant difference in age, size and location of RAO vs non rt-associated osarc. Overall, all osarc pts with metastatic disease at diagnosis fared poorly. Pts presenting with localized RAO had worse outcomes than patients with localized SO. This was not associated with a detectable difference in therapy rendered or treatment effect in resection specimens. [Table: see text]

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Safety and Efficacy of Venetoclax-Based Treatment in Elderly CLL Patients: A Retrospective Study from the Filo Group

Blood ◽

10.1182/blood-2021-149640 ◽

2021 ◽

Vol 138 (Supplement 1) ◽

pp. 3747-3747

Author(s):

Charlotte Doublet ◽

Marie-Sarah Dilhuydy ◽

Emmanuelle Ferrant ◽

Pierre Feugier ◽

Alexandra Fayault ◽

...

Keyword(s):

Retrospective Study ◽

Board Of Directors ◽

Research Funding ◽

Progression Free Survival ◽

Oncology Nurses ◽

Advisory Committees ◽

Free Survival ◽

Daily Dose ◽

History Of

Abstract Median age at diagnosis of chronic lymphocytic leukemia is 72 years. However, only few patients over 80 years of age are included in clinical trials, even in those devoted to unfit patients. In order to evaluate both efficiency and safety of venetoclax in this category of patients, we conducted a multicentric retrospective study and collected data from 77 CLL patients from 19 FILO centers who started venetoclax after 80 years of age. Median age at venetoclax initiation was 86 years old (81-97). 63% of patients had a history of heart disease, 62% had renal failure (moderate 59% and severe 3%) and 29% had a history of severe infections. Despite their comorbidities and a CIRS greater than 6 in 70% of cases, their autonomy was preserved with a median performans status of 1 (0-4). In this comorbid geriatric population, pretherapeutic geriatric assessment was only performed in a single patient. The median number of prior therapies was 2 (0-6) with an exposure to a BCR inhibitor in 56% of cases. 11q and 17p deletion were found in 39% and 30% of cases respectively, 39% of patients had a complex karyotype and 30% harbored a TP53 mutation. However, in this real life population, these prognostic factors were only performed in half of patients. IGHV mutational status was only available in 11 patients, and 83% of them had unmutated IGHV. At the time of venetoclax initiation, the tumor lysis syndrome (TLS) risk was moderate in 57% of cases and high in 8% of cases. Venetoclax was administered as a single agent (42%) or in association with rituximab (58%). In total, half of the patients were hospitalized at each dose ramp-up, and only 3 patients were treated on outpatient basis. 82% of the cohort was able to reach the daily dose of 400mg. Half of the patients were included in a phone call monitoring program with oncology nurses to pre-emptively manage side effects and foster therapy adherence. The safety study reported 14% of TLS, with 2 discontinuations of treatment within the first month: one of which led to dialysis and the other to death. As in the previously published studies, 25% of patients had infectious complications, and grade 3 haematological and digestive toxicities were reported in 42% and 22% of cases, respectively. The reduction of the daily dose of venetoclax was necessary for 33%. Permanent discontinuation of venetoclax occurred in 40% of subjects, including 29% of early withdrawal (within the first 3 months). Main reasons for discontinuation were intolerance (21%), CLL progression (21%), death (21%) and scheduled treatment discontinuation (10%). The overall response rate was 86%, consisting of 49% of complete response (unconfirmed by bone marrow biopsy) and 37% of partial response. With a median follow-up of 21months, estimated progression free survival and overall survival were 29 and 38 months respectively. Prior exposure to a BCR inhibitor had no impact on progression free survival. To conclude, venetoclax has a manageable safety profile in elderly patients with comorbidities and can induce prolonged responses. Finally, if additional follow-up by oncology nurses seems to be more and more implemented, the pre-therapeutic onco-geriatric evaluation remains underexploited in this population. Disclosures Ferrant: AstraZeneca: Honoraria; AbbVie: Honoraria, Other: Travel, Accommodations, Expenses; Janssen: Other: Travel, Accommodations, Expenses. Feugier: Janssen: Consultancy, Honoraria; Gilead: Consultancy, Honoraria; Abbvie: Consultancy, Honoraria; Amgen: Honoraria; Astrazeneca: Consultancy, Honoraria. Laribi: AstraZeneca: Other: Personal Fees; Le Mans Hospital: Research Funding; AbbVie: Other: Personal Fees, Research Funding; Jansen: Research Funding; Novartis: Other: Personal Fees, Research Funding; IQONE: Other: Personal Fees; Astellas Phama, Inc.: Other: Personal Fees; BeiGene: Other: Personal Fees; Takeda: Other: Personal Fees, Research Funding. Tchernonog: JANSSEN: Consultancy; ABBVIE: Consultancy; ASTRAZENECA: Consultancy. Dartigeas: Astra-Zeneca: Membership on an entity's Board of Directors or advisory committees, Other: travel grants/Congress; Abbvie: Membership on an entity's Board of Directors or advisory committees, Other: travel grants/Congress; Janssen: Membership on an entity's Board of Directors or advisory committees, Other: travel grants/Congress. Quinquenel: Abbvie: Honoraria; Janssen: Honoraria; AstraZeneca: Honoraria.

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Combined Treatment of Bortezomib, Continuous Low-Dose Cyclophosphamide and Dexamethasone Followed By One Year of Maintenance Treatment for Refractory or Relapsed Multiple Myeloma Patients: A Prospective Phase II Study

Blood ◽

10.1182/blood.v124.21.4733.4733 ◽

2014 ◽

Vol 124 (21) ◽

pp. 4733-4733

Author(s):

Esther GM Waal de ◽

Linda Munck de ◽

Gerhard Woolthuis ◽

Annet velden Van Der ◽

Yvonne Tromp ◽

...

Keyword(s):

Overall Survival ◽

Maintenance Therapy ◽

Research Funding ◽

Low Dose ◽

Progression Free Survival ◽

Free Survival ◽

First Relapse ◽

Grade 3 ◽

One Year

Abstract Introduction: Combination therapy for longer periods but at low dose, also called metronomic scheduling, might be an effective manner to treat patients with relapsing myeloma. In particular if the used agents attack the malignant clone in an alternative manner. Therefore we used the combination of bortezomib, dexametasone and daily low dose of oral cyclophosphamide as an induction regimen followed by one year of maintenance therapy consisting of bortezomib and cyclophosphamide. Methods: Relapsing myeloma patients, bortezomib naïve, were treated with three cycles of 1.3 mg/m2 bortezomib at day 1, 4, 8 and 11, cyclophosphamide 50 mg daily, and 20 mg dexamethasone at day 1, 2, 4, 5, 8, 9, 11 and 12 followed by three cycles of bortezomib 1.6 mg/m2 (day 1, 8, 15 and 2), cyclophosphamide (50 mg) daily and dexamethasone (20 mg) at day 1, 2, 8, 9, 15, 16, 22 and 23. Maintenance therapy consisting of bortezomib 1.3 mg/m2 every two weeks and daily dose of 50 mg cyclophosphamide for one year was applied to patients in partial or complete remission. Primary endpoints were toxicity during re-induction and maintenance therapy. Secondary endpoints were response to treatment and progression free and overall survival. Results: 59 patients with relapsing multiple myeloma were included of whom 69% were in first relapse (Table 1). The upfront treatment consisted mainly of thalidomide-based and vincristine-based chemotherapy and 40% of the patients have been treated with an autologous stem cell transplantation. All 6 cycles of induction chemotherapy could be given in 49% of the patients. Premature discontinuation before starting maintenance therapy was due to toxicity (31%), progressive disease (7%), death (7%) or other reasons (6%). Myelosuppression was the most common side effect with WHO grade 3-4 in 31% of the patients. Neuropathy grade 3-4 was observed in 16% of patients, partially due to the fact that bortezomib was given intravenously during the first 2 yrs of the protocol which included 76% of the patients. Maintenance therapy was started in 47% of the patients with a median duration of 7.3 months (range 0.36.-13.4). Grade 3-4 toxicity was observed in 25% of the patients including infections (n=3) and myelosuppression (n=3) which did not resulted in discontinuation of therapy. Median follow up time was 29 months with an overall response of 62%, and a very good partial response (VGPR), complete remission (CR) in 21% and 7% of the patients respectively. During the maintenance phase an improvement in responsiveness was observed in 25% of the patients. The CR rate increased with 9% to a total of 16%. VGPR rate was 20% and 16% of the patient had a PR. At end of the maintenance therapy 50% of patients started with maintenance had stable disease. The median progression free survival (PFS) was 17.2 months (range 0.13 – 43.5) as depicted in figure 1. and the median overall survival was 21.6 months (range 0.46-54.4, figure 2). During follow up 33 % of the patients died due to progression of MM. Conclusion: The present study demonstrates that combination therapy with bortezomib, continuous low dose cyclophosphamide and dexamethasone is an effective and manageable regimen. Adding a year of maintenance was feasible with limited side effects and an increase in CR rate. Table 1: patient characteristics Patients (%) Age, mean (min,max) 69 (46-86) Sex Male 56 Female 44 Relapse number First relapse 75 Second relapse 20 Third relapse 5 Performance status 0 65 1 29 2 5 M-protein heavy chain IgA 18 IgG 65 Light chain disease 18 Polyneuropathy No 61 Yes 39 Figure 1: Progression free survival Figure 1:. Progression free survival Figure 2: Overall survival Figure 2:. Overall survival Disclosures Waal de: Jansen Cilag: Research Funding. Munck de:Jansen Cilag: Research Funding. Woolthuis:Jansen Cilag: Research Funding. velden Van Der:Jansen Cilag: Research Funding. Tromp:Jansen Cilag: Research Funding. Hoogendoorn:Jansen Cilag: Research Funding. Vellenga:Jansen Cilag: Research Funding. Hovenga:Jansen Cilag: Research Funding.

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Early radiotherapy versus late radiotherapy after incomplete transarterial chemoembolization in hepatocellular carcinoma.

Journal of Clinical Oncology ◽

10.1200/jco.2015.33.3_suppl.459 ◽

2015 ◽

Vol 33 (3_suppl) ◽

pp. 459-459

Author(s):

Mi Sun Kim ◽

Do Young Kim ◽

Kwang-Hyub Han ◽

Jong Yun Won ◽

Do Yun Lee ◽

...

Keyword(s):

Multivariate Analysis ◽

Treatment Group ◽

Survival Rates ◽

Three Dimensional ◽

Evaluation Criteria ◽

Progression Free Survival ◽

Free Survival ◽

Vessel Invasion ◽

One Year ◽

The One

459 Background: To investigate the efficacy of early RT (ERT) in comparison to late RT (LRT) in HCC patients after incomplete TACE. Methods: Between January 2006 and December 2011, 99 BCLC B and C stage HCC patients were reviewed. We defined ERT as curative aimed additional RT after the first TACE, and LRT was defined as salvage aimed additional RT for recurrent or re-growing remnant tumors after repeated TACE. The median prescribed dose was 45 Gy (range, 21.6 Gy–60 Gy) given in daily dose of 1.8 Gy–3 Gy. Three-dimensional conformal RT or intensity modulated RT was used for all patients. The in-field tumor response rate (IFTR) was evaluated using the modified response evaluation criteria in solid tumors. Results: Fifty eight (58.6%) patients received ERT and 41 (41.4%) patients received LRT. All patients had BCLC B-C stage HCC and the characteristics between two groups were not different significantly. However, vessel invasion was observed more often in the LRT group (p=0.039). The median interval between last TACE and RT was 2weeks in the ERT group, and 4 weeks in the LRT group. The IFTR at one month and three months were significantly higher in the ERT group. The one-year in-field progression-free survival rates were 79.5% and 45.9% in the ERT and LRT groups, respectively (p=0.007).The one-year overall survival rates were 75.9% and 48.8% in the ERT and LRT groups, respectively (p = 0.01). In the multivariate analysis, vessel invasion and treatment group (ERT vs.LRT) were independent predictor of OS (p=0.039 and p=0.035). PVTT and treatment group (ERT vs.LRT) showed correlation with IFPFS in the multivariate analysis (p=0.002 and p=0.01). Conclusions: While this finding may reflect differences in biological characteristics between the two groups, ERT within 2weeks after incomplete TACE should be considered.

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Clinical Application of Autologous Adipose Stem Cells in Patients with Multiple Sclerosis: Preliminary Results

Mediators of Inflammation ◽

10.1155/2016/5302120 ◽

2016 ◽

Vol 2016 ◽

pp. 1-5 ◽

Cited By ~ 13

Author(s):

Adam Stepien ◽

Natalia L. Dabrowska ◽

Marzena Maciagowska ◽

Renata Piusinska Macoch ◽

Aleksandra Zolocinska ◽

...

Keyword(s):

Multiple Sclerosis ◽

Disease Progression ◽

Progression Free Survival ◽

Entire Group ◽

Adipose Stem Cell ◽

Free Survival ◽

Inflammatory Phase ◽

Efficacy Measures ◽

One Year

The clinical outcome of autologous adipose stem cell (ASC) treatment of patients with multiple sclerosis (MS) was investigated following one year of observation.Methods. The clinical and MRI outcomes of 16 ASC-treated patients with RRMS and SPMS are reported after a one-year follow-up period.Results.At 18 months of follow-up, some patients showed “enticing” improvements on some exploratory efficacy measures, although a significant benefit was not observed for any measure across the entire group. Neither the progression of disability nor relapses were observed in any cases. In four patients, we found new gadolinium+ (Gd+) lesions on MRI. Our results indicate that ASC therapy is safe and does not produce any substantial side effects. Disease progression-free survival (PFS) of 18 months was seen in all patients with RRMS and SPMS. In these patients, EDSS scores did not progress above baseline scores. Gd-enhancing lesions were observed in two cases with RRMS, but these patients did not exhibit changes in EDSS score.Conclusion. Intrathecal treatment with ASCs is an attractive form of therapy for patients with MS but should be reserved for cases with aggressive disease progression, for cases that are still in the inflammatory phase, and for the malignant form.

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Primary hypertension to predict progression-free survival of target therapy in patients with metastatic renal cell carcinoma.

Journal of Clinical Oncology ◽

10.1200/jco.2012.30.15_suppl.e15052 ◽

2012 ◽

Vol 30 (15_suppl) ◽

pp. e15052-e15052

Author(s):

Zhi Hong Chi ◽

Li Li Mao ◽

Lu Si ◽

Chuan Liang Cui ◽

Xi Nan Sheng ◽

...

Keyword(s):

Blood Pressure ◽

Renal Cell Carcinoma ◽

Targeted Therapy ◽

Cell Carcinoma ◽

Renal Cell ◽

Progression Free Survival ◽

Primary Hypertension ◽

Free Survival ◽

History Of

e15052 Background: Primary Hypertension(PH) was identified as a risk factor for RCC(renal cell carcinoma) in previous studies, only one report showed history of hypertension predicted better survival in RCC patients. In recent years, treatment related hypertension (TRH) was found to be associated with clinical outcome of targeted therapy. However, little is known about whether primary hypertension had an effect on survival of mRCC patients treated with targeted therapy. The objective of this study was to investigate the effects of primary hypertension on treatment outcome in patients treated with targeted therapy for metastatic RCC (mRCC). Methods: We conducted follow-up on a cohort of consecutiave mRCC cases treated with sunitinib or sorafenib in our center. Baseline characters were recorded such as age, gender, blood pressure, stage, et al. All the patients were followed up for progression free survival (PFS), overall survival (OS), objective response rate(RR). Results: We studied a cohort of 77 cases, 58(24.7%) pts were male, 18(23.4%) pts were 65 years or older, and 47 (61%) pts received sunitinib. 28 pts(36.4%) had a history of PH and 19 pts (24.7%) developed TRH, which was more common in PH pts than pts with normal blood pressure(p=0.003). With a median follow-up time of 24 months (range: 1.5-36.2 months), 60 pts developed disease progression and 34 pts died. Patients with primary hypertension demonstrated longer median PFS than those with normal baseline blood pressure (14.0m vs 9.5m, 95%CI: 8.0-10.9m) (p=0.01). In multivariable analysis, primary hypertension was independent predictors of PFS (HR =0.51, 95% CI; 0.27-0.98). No significant differences were found between clinical outcomes and other factors such as gender, age. Conclusions: Data from this study suggested that primary hypertension may be an independent predictor of longer PFS in mRCC pts treated with targeted agents.

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Time to central nervous system (CNS) metastases (mets) with atezolizumab (A) or placebo (P) combined with cobimetinib (C) + vemurafenib (V) in the phase III IMspire150 study.

Journal of Clinical Oncology ◽

10.1200/jco.2020.38.15_suppl.10023 ◽

2020 ◽

Vol 38 (15_suppl) ◽

pp. 10023-10023 ◽

Cited By ~ 2

Author(s):

Paolo Antonio Ascierto ◽

Caroline Robert ◽

Karl D. Lewis ◽

Rodrigo Munhoz ◽

Gabriella Liszkay ◽

...

Keyword(s):

Cumulative Incidence ◽

Survival Rates ◽

Progression Free Survival ◽

Tumor Burden ◽

Phase Iii ◽

Sensitivity Analyses ◽

Free Survival ◽

History Of ◽

To Receive

10023 Background: The phase 3 IMspire150 study (NCT02908672) demonstrated improved progression-free survival with first-line combination treatment with A+C+V vs P+C+V in patients (pts) with previously untreated BRAFV600 mutation–positive advanced melanoma. Here we report incidence and time to development of CNS mets with A+C+V vs P+C+V in the IMspire150 study. Methods: Eligible pts were randomized 1:1 to receive A+C+V or P+C+V. A or P were given on day 1 and 15 of each 28-day cycle after an initial cycle of C+V. Incidence and time to development of CNS mets were evaluated in pts with no history of CNS mets at baseline confirmed by magnetic resonance imaging/computed tomography (MRI/CT). On study MRI/CT assessments were performed as clinically indicated. Time-to-event outcomes were estimated using the Kaplan-Meier method and competing risks analysis. Sensitivity analyses were conducted using landmark analysis at time of initiation of A or P. Results: 514 pts were randomized to receive A+C+V (n = 256) or P+C+V (n = 258); 244 and 247 pts, respectively, had no history of CNS mets at baseline. After a median follow-up of 18.9 months, CNS mets had developed in 52/244 pts (21%) in the A+C+V arm and 62/247 pts (25%) in the P+C+V arm. In both arms, pts with CNS mets were more likely to have other known unfavorable prognostic factors: elevated LDH, presence of liver mets, and/or higher tumor burden. Cumulative incidence of CNS mets as first site of progressive disease with A+C+V vs P+C+V was 8% vs 9%, 16% vs 19%, 20% vs 24%, and 23% vs 26% at 6, 12, 18, and 24 months, respectively (hazard ratio [HR] 0.87; 95% CI 0.60-1.26). Estimated CNS mets-free survival rates for A+C+V vs P+C+V were 91% vs 90%, 81% vs 75%, 74% vs 66%, and 69% vs 62% at 6, 12, 18, and 24 months, respectively, with a trend for improved CNS mets-free survival with A+C+V (HR 0.79; 95% CI 0.55-1.14). Results of landmark analyses for CNS mets-free survival and cumulative incidence of CNS mets were similar to those in the overall analysis. Conclusions: The addition of anti–programmed death-ligand 1 to C+V is associated with numerically lower rates of interval development of CNS mets, consistent with the overall benefit observed for A+C+V in the study. This finding requires further follow-up to fully assess the magnitude of benefit of A+C+V on CNS mets-free survival. Clinical trial information: NCT02908672.

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Impact of Standardized Allergen-RemovedRhus vernicifluaStokes Extract on Advanced Adenocarcinoma of the Ampulla of Vater: A Case Series

Evidence-based Complementary and Alternative Medicine ◽

10.1155/2013/203168 ◽

2013 ◽

Vol 2013 ◽

pp. 1-8 ◽

Cited By ~ 7

Author(s):

Woncheol Choi ◽

Soomin An ◽

Eunmi Kwon ◽

Wankyu Eo ◽

Sanghun Lee

Keyword(s):

Evaluation Criteria ◽

Case Series ◽

Progression Free Survival ◽

Final Analysis ◽

Complete Response ◽

Ampulla Of Vater ◽

Free Survival ◽

Rhus Verniciflua ◽

Rare Malignancy

Background. Adenocarcinoma of the ampulla of Vater (AAV) is a rare malignancy that has a better prognosis than other periampullary cancers. However, the standard treatment for patients with relapsed or metastatic AAV has not been established. We investigated the clinical feasibility of standardized allergen-removedRhus vernicifluastokes (aRVS) extract for advanced or metastatic AAV.Patients and Methods. From July 2006 to April 2011, we retrospectively reviewed all patients with advanced AAV treated with aRVS extract alone. After applying inclusion/exclusion criteria, 12 patients were eligible for the final analysis. We assessed the progression-free survival (PFS) and overall survival (OS) of these patients during the follow-up period.Results. The median aRVS administration period was 147.0 days (range: 72–601 days). The best tumor responses according to Response Evaluation Criteria in Solid Tumors were as follows: two with complete response, two with stable disease, and eight with progressive disease. The median OS was 15.1 months (range: 4.9–25.1 months), and the median PFS was 3.0 months (range: 1.6–11.4 months). Adverse reactions to the aRVS treatment were mostly mild and self-limiting.Conclusions. Prolonged survival was observed in patients with advanced AAV under the treatment of standardized aRVS extract without significant adverse effects.

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Management and outcome of metastatic pheochromocytomas/paragangliomas: a monocentric experience

Journal of Endocrinological Investigation ◽

10.1007/s40618-021-01629-x ◽

2021 ◽

Author(s):

G. De Filpo ◽

G. Cantini ◽

G. Rastrelli ◽

G. Vannini ◽

T. Ercolino ◽

...

Keyword(s):

Multidisciplinary Approach ◽

Progression Free Survival ◽

Initial Diagnosis ◽

University Hospital ◽

Inherited Disease ◽

Biochemical Activity ◽

Free Survival ◽

Sporadic Disease ◽

Worse Prognosis

Abstract Background Pheochromocytoma (PHEO) and paraganglioma (PGL) are rare neuroendocrine tumors releasing catecholamines. Metastatic pheochromocytomas/paragangliomas (PPGLs) occur in about 5–26% of cases. To date, the management of patients affected by metastatic disease is a challenge in the absence of guidelines. Aim The aim of this study was to evaluate the overall survival (OS) and the progression-free survival (PFS) in metastatic PPGLs. Methods Clinical data of 20 patients referred to the Careggi University Hospital (Florence, Italy) were retrospectively collected. Follow-up ranged from 1989 to 2019. Site and size of primary tumor, biochemical activity, genetic analysis and employed therapies were considered. Data were analyzed with SPSS version 27. Results Nine PHEOs (45%) and 11 PGLs (55%) were enrolled. Median age at diagnosis was 43.5 years [30–55]. Mean follow-up was 104.6 ± 89.3 months. Catecholamines were released in 70% of cases. An inherited disease was reported in 50% of patients. OS from the initial diagnosis (OSpt) and from the metastatic appearance (OSmtx) were lower in older patients (OSpt p = 0.028; OSmtx p < 0.001), abdominal PGLs (OSpt p = 0.007; OSmtx p = 0.041), larger tumors (OSpt p = 0.008; OSmtx p = 0.025) and sporadic disease (OSpt p = 0.013; OSmtx p = 0.008). Conclusion Our data showed that older age at the initial diagnosis, sympathetic extra-adrenal localization, larger tumors and wild-type neoplasms are related to worse prognosis. Notably, the employed therapies do not seem to influence the survival of our patients. At present, effective treatments for metastatic PPGLs are missing and a multidisciplinary approach is indispensably required.

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