scholarly journals Metastatic Pheochromocytomas and Abdominal Paragangliomas

2021 ◽  
Author(s):  
Dan Granberg ◽  
Carl Christofer Juhlin ◽  
Henrik Falhammar
Keyword(s):  
Metastatic Potential ◽  
Somatostatin Analogues ◽  
Primary Treatment ◽  
Treatment Modalities ◽  
Line Treatment ◽  
First Line ◽  
Biochemical Tests ◽  
Pubmed Database ◽  
Tumor Boards ◽  
131I Mibg

Abstract Context Pheochromocytomas and paragangliomas (PPGLs) are believed to harbor malignant potential; about 10% to 15% of pheochromocytomas and up to 50% of abdominal paragangliomas will exhibit metastatic behavior. Evidence Acquisition Extensive searches in the PubMed database with various combinations of the key words pheochromocytoma, paraganglioma, metastatic, malignant, diagnosis, pathology, genetic, and treatment were the basis for the present review. Data Synthesis To pinpoint metastatic potential in PPGLs is difficult, but nevertheless crucial for the individual patient to receive tailor-made follow-up and adjuvant treatment following primary surgery. A combination of histological workup and molecular predictive markers can possibly aid the clinicians in this aspect. Most patients with PPGLs have localized disease and may be cured by surgery. Plasma metanephrines are the main biochemical tests. Genetic testing is important, both for counseling and prognostic estimation. Apart from computed tomography and magnetic resonance imaging, molecular imaging using 68Ga-DOTATOC/DOTATATE should be performed. 123I-MIBG scintigraphy may be performed to determine whether 131I-MIBG therapy is a possible option. As first-line treatment in patients with metastatic disease, 177Lu-DOTATATE or 131I-MIBG is recommended, depending on which shows best expression. In patients with very low proliferative activity, watch-and-wait or primary treatment with long-acting somatostatin analogues may be considered. As second-line treatment, or first-line in patients with high proliferative rate, chemotherapy with temozolomide or cyclophosphamide + vincristine + dacarbazine is the therapy of choice. Other therapies, including sunitinib, cabozantinib, everolimus, and PD-1/PDL-1 inhibitors, have shown modest effect. Conclusions Metastatic PPGLs need individualized management and should always be discussed in specialized and interdisciplinary tumor boards. Further studies and newer treatment modalities are urgently needed.

scholarly journals Efficacy of pasireotide in controlling severe hypercortisolism until cardiac transplantation

2017 ◽  
Vol 2017 ◽  
Author(s):  
Roberto Attanasio ◽  
Liana Cortesi ◽  
Daniela Gianola ◽  
Claudia Vettori ◽  
Fulvio Sileo ◽  
...  
Keyword(s):  
Heart Transplantation ◽  
Cushing’S Syndrome ◽  
Cardiac Transplantation ◽  
Serum Cortisol ◽  
Primary Treatment ◽  
Cushing's Syndrome ◽  
Line Treatment ◽  
First Line ◽  
First Line Treatment ◽  
Undergo Heart Transplantation

Summary Cushing’s syndrome is associated with increased morbidity and mortality. Although surgery is the first-line treatment, drugs can still play a role as an ancillary treatment to be employed while waiting for surgery, after unsuccessful operation or in patients unsuitable for surgery. We were asked to evaluate a 32-year-old male waiting for cardiac transplantation. Idiopathic hypokinetic cardiomyopathy had been diagnosed since 6 years. He was on treatment with multiple drugs, had a pacemaker, an implantable cardioverter and an external device for the support of systolic function. Physical examination showed severely impaired general status, signs of hypercortisolism and multiple vertebral compression fractures. We administered teriparatide, and the few evaluable parameters supported the diagnosis of ACTH-dependent hypercortisolism: serum cortisol was 24.2 µg/dL in the morning and 20.3 µg/dL after overnight 1 mg dexamethasone, urinary free cortisol (UFC) was 258 µg/24 h and ACTH 125 pg/mL. Pituitary CT was negative. Pasireotide 300 µg bid was administered and uptitrated to 600 µg bid. Treatment was well tolerated, achieving dramatic improvement of clinical picture with progressive normalization of serum cortisol and ACTH levels as well as UFC. After 4 months, the patient underwent successful heart transplantation. Many complications ensued and were overcome. Pituitary MRI was negative. On pasireotide 300 µg bid and prednisone 2.5 mg/day (as part of immunosuppressive therapy), morning serum cortisol and ACTH were 15.6 µg/dL and 54 pg/mL respectively, UFC was 37 µg/24 h, fasting glucose: 107 mg/dL and HbA1c: 6.5%. In conclusion, primary treatment with pasireotide achieved remission of hypercortisolism, thus allowing the patient to undergo heart transplantation. Learning points: Untreated Cushing’s syndrome is associated with ominous prognosis. First-line treatment is surgery (at pituitary or adrenal, according to disease localization). A few drugs are available to treat hypercortisolism. Pasireotide is a multi-ligand somatostatin analog approved for treatment of hypercortisolism. Primary treatment with pasireotide was effective in a patient with severe Cushing’s syndrome, allowing him to undergo heart transplantation.

scholarly journals Selecting the first line treatment in non-metastatic hepatocellular carcinoma - comparing clinical practice guidelines

2020 ◽  
Vol 14 (2) ◽  
Author(s):  
Soumya Jogi ◽  
Radha Varanai ◽  
Sravani S. Bantu ◽  
Ashish Manne
Keyword(s):  
Hepatocellular Carcinoma ◽  
Clinical Practice ◽  
Systemic Therapy ◽  
Residual Disease ◽  
Disease Process ◽  
Locoregional Therapy ◽  
Line Treatment ◽  
First Line ◽  
Tumor Boards ◽  
First Line Treatment

Primary malignancy of the liver or hepatocellular carcinoma (HCC) is unique in its presentation, disease process, and management. Unlike breast or colon cancer, the staging of HCC depends on performance status and baseline liver function along with pathological characteristics. Apart from traditional options like surgery and systemic therapy, effective management can be achieved in selected cases with liver transplant and locoregional therapy (LRT) like transarterial chemoembolization (TACE), transarterial radioembolization (TARE), and ablation. Liver study societies and cancer groups across the globe proposed guidelines to aid the treating physicians in choosing first-line treatment for liver cancer. It is tough to compare these guidelines as they differ not only in treatment recommendations but also in risk assessment (and staging). The approach to the same patient may be different in the country he or she is managed. In clinical practice, decisions are usually taken on the consensus of multidisciplinary tumor boards and do not necessarily adhere to any guidelines. In the early (and very early) stage HCC, curative options like surgery, transplant, and ablation are recommended. In intermediate stage HCC, LRT (TACE and TARE) is preferred in the first line and systemic therapy for treatment failure or residual disease. Systemic therapy, including the atezolizumab/bevacizumab combination and tyrosine kinase inhibitors (TKI) like sorafenib and lenvatinib, is used for advanced stages. Supportive care is advised for terminal stage HCC.

scholarly journals Management of Pearly Penile Papules: A Review of the Literature

2019 ◽  
Vol 24 (1) ◽  
pp. 79-85 ◽  
Author(s):  
Anthony D. Honigman ◽  
Danielle P. Dubin ◽  
Justin Chu ◽  
Matthew J. Lin
Keyword(s):  
Psychological Distress ◽  
Long Term Results ◽  
Treatment Modalities ◽  
Line Treatment ◽  
Review Of The Literature ◽  
First Line ◽  
Cosmetic Outcomes ◽  
First Line Treatment ◽  
Ablative Laser

Pearly penile papules (PPPs) are benign, dome-shaped lesions found around the corona of the penis. Despite being asymptomatic and benign in nature, the appearance of PPPs may cause a great deal of psychological distress to both the patient and their sexual partner. While patient reassurance may be the first-line treatment, several other treatment modalities including cryotherapy, electrodessication and curettage, and laser therapy have all been used to treat PPPs in order to achieve a cosmetic outcome that satisfies the patient. Based on the evaluation of the existing literature, ablative laser therapies offer satisfactory cosmetic outcomes with good long-term results.

Prognostic factors and first-line treatment modalities in nonagenarian patients with lung cancer

2019 ◽  
Vol 10 (3) ◽  
pp. 439-441
Author(s):  
Cheng-Chieh Hsu ◽  
Ying-Tzu Huang ◽  
Yen-Han Tseng ◽  
Yung-Hung Luo ◽  
Yuh-Min Chen
Keyword(s):  
Lung Cancer ◽  
Prognostic Factors ◽  
Treatment Modalities ◽  
Line Treatment ◽  
First Line ◽  
First Line Treatment

scholarly journals Failure to Achieve CR with First-Line Treatment Is the Primary Cause of Treatment Failure in T-Cell Lymphoma: The Princess Margaret Cancer Centre Experience

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 3005-3005
Author(s):  
Umberto Falcone ◽  
Melania Pintilie ◽  
Ri Wang ◽  
Vishal Kukreti ◽  
John G. Kuruvilla ◽  
...  
Keyword(s):  
T Cell ◽  
Cell Lymphoma ◽  
Primary Treatment ◽  
T Cell Lymphoma ◽  
Line Treatment ◽  
First Line ◽  
Limited Stage ◽  
Stage Disease ◽  
First Line Treatment

Abstract Introduction: T-cell lymphomas (T-NHL) represent rarer entities compared to B-NHL, accounting for 5% -10% of NHL in Western countries and 15%-20% in Asia. They are divided into clinico-pathologic subtypes based on etiology, morphology, and clinical behavior. Because of the rarity and the lack of specific histologic features for the different subtypes, the diagnosis is difficult and clinical picture is usually very helpful to establish the diagnosis. We conducted a retrospective analysis of patients (pts) with T-NHL treated at our Centre, with the purpose of studying overall outcome and possible prognostic factors, including histologic subtypes, from our database. Patients and methods: Consecutive T-NHL pts (excluding Adult T-cell leukemia/lymphoma, NK/T NHL, and primary cutaneous T-NHL), receiving primary treatment at the Princess Margaret Cancer Centre (PMCC) between 2001-2014 were included. Data were extracted from a prospective patient database and the medical record regarding baseline characteristics, treatment, response and outcome. Response assessment was with CT imaging as per 1999 Working Group criteria. Results: Of a total of 2155 pts with aggressive histology NHL treated at PMCC between 2001-2014, 2031 pts had B-NHL and 124 (5.7%) T-NHL. Median age was 56 years (18-90), male/female ratio: 2.4; 63% presented with advanced stage (III-IV) disease, 22% had bone marrow involvement; 63% had elevated LDH and 44% had B symptoms. Observed subtypes were: Peripheral T-cell lymphoma, NOS 58 pts (PTCL NOS, 47%), Anaplastic large cell lymphoma, ALK-negative 16 pts (ALCL-ALK-, 13%), ALCL, ALK-positive 22 pts (ALCL-ALK+, 18%), Angioimmunoblastic T-cell lymphoma 13 pts (AITL, 10.5%), Enteropathy-associated T-cell lymphoma 7 pts (EATL, 5.6%), Hepatosplenic T-cell lymphoma 8 pts (HSTCL, 6%). 105/124 pts (85%) received induction chemotherapy; CHOP-like regimens were used in 94 pts (90%), and involved field radiation therapy (RT) was included in primary treatment in 24 pts (19%). Nineteen pts were treated palliatively, 5 pts with RT alone, 14 pts received palliative chemotherapy or supportive care only. Complete response (CR) was obtained in 63/105 pts (60%; Table 2), PR in 2 pts (1.9%) and 40 pts had no response or progressive disease (SD and PD; 38%). Considering together the most common subtypes (ALCL-ALK+/-, AITL, PTCL NOS), CR rate was 84% in limited stage vs 52% in advanced stage disease. Among patients with CR, 24 relapsed (38%). Fourteen pts received autologous stem cell transplant (8 at relapse, 6 for PD); 7/14 (50%) were alive at last follow-up. At a median follow-up of 5.3 years, 57/124 (46%) pts are alive. Cause of death was T-NHL in 50/124 (40%) pts. Two pts died of second malignancy (1.6%). Median overall survival (OS) and progression-free survival (PFS) were 4.57 years (95%CI: 2.23-9.53; 5yOS: 48%) and 1.5 years (0.87-3.17; 5yPFS: 37%), respectively (Table 2). For pts with limited stage disease median PFS was 4.57 years (5yPFS: 49%) and median OS 10.0 years (5yOS: 62%), while for pts with stage III/IV, median PFS was 0.82 years (5yPFS: 31%) and OS 1.81 years (5yOS: 38%). Median OS for pts who did not experience relapse (39/63; 62%) was 13.38 years (95%CI: 9.53-NA; 5yOS: 93%) vs 3.12 years (95%CI: 1.69-4.07 years; 5yOS: 25%) in pts who had relapse after CR1 (p<0.001). Pts failing to achieve CR (PR, SD, PD) had a very poor outcome with median OS 1.15 years (95%CI: 0.62-1.32 years; 5yOS: 17%). For PTCL NOS pts, outcomes were poor while those with ALCL had more favorable results regardless of ALK status (Table 2). Conclusions: Failure to achieve CR with first-line treatment was the main cause of treatment failure in patients with T-NHL treated with anthracycline-based chemotherapy. Patients with limited stage lymphoma and with ALCL ALK+/- subtypes have more favorable outcomes. For PTCL NOS, the most common subtype, and less common entities (HSTCL, EATL), results are poor and new induction strategies are needed. Disclosures Kukreti: Celgene: Honoraria; Amgen: Honoraria; Lundbeck: Honoraria.

scholarly journals Sequence of therapy and survival in patients with advanced pancreatic neuroendocrine tumours

2020 ◽  
Vol 27 (4) ◽  
Author(s):  
E. S. Tsang ◽  
J.M. Loree ◽  
C. Speers ◽  
H.F. Kennecke
Keyword(s):  
Neuroendocrine Tumours ◽  
Progression Free Survival ◽  
Somatostatin Analogues ◽  
Treatment Modalities ◽  
Primary Role ◽  
First Line ◽  
Optimal Sequence ◽  
Systemic Treatments ◽  
Pancreatic Neuroendocrine Tumours ◽  
Line Of Therapy

Background Pancreatic neuroendocrine tumours (pnets) often present as advanced disease. The optimal sequence of therapy is unknown. Methods Sequential patients with advanced pnets referred to BC Cancer between 2000 and 2013 who received 1 or more treatment modalities were reviewed, and treatment patterns, progression-free survival (pfs), and overall survival (os) were characterized. Systemic treatments included chemotherapy, small-molecule therapy, and peptide receptor radiotherapy. Results In 66 cases of advanced pnets, median patient age was 61.2 years (25%–75% interquartile range: 50.8–66.2 years), and men constituted 47% of the group. First-line therapies were surgery (36%), chemotherapy (33%), and somatostatin analogues (32%). Compared with first-line systemic therapy, surgery in the first line was associated with increased pfs and os (20.6 months vs. 6.3 months and 100.3 months vs. 30.5 months respectively, p < 0.05). In 42 patients (64%) who received more than 1 line of therapy, no difference in os or pfs between second-line therapies was observed. Conclusions Our results confirm the primary role of surgery for advanced pnets. New systemic treatments will further increase options.

Targeted therapy in gastrointestinal stromal tumor (GIST): A nationwide database analysis from Taiwan.

2014 ◽  
Vol 32 (3_suppl) ◽  
pp. 437-437
Author(s):  
I-Jen Chiang ◽  
Wen-Yi Shau ◽  
Wei-Tse Fang ◽  
Chi-Hui Fang ◽  
Yen-Yang Chen
Keyword(s):  
Targeted Therapy ◽  
Gastrointestinal Stromal Tumor ◽  
Dose Escalation ◽  
Targeted Therapies ◽  
Standard Dose ◽  
Stromal Tumor ◽  
Treatment Modalities ◽  
Research Database ◽  
Line Treatment ◽  
First Line

437 Background: Gastrointestinal stromal tumor (GIST) is a visceral sarcoma that arises from the gastrointestinal tract. Currently, imatinib (first-line) and sunitinib (second-line) are two reimbursed targeted therapies for GIST in Taiwan. This study aimed to evaluate the real world data of targeted therapies in GIST treatment among Taiwanese population. Methods: We conducted a nationwide retrospective cohort study based on data from the National Health Insurance Research Database (NHIRD) between January 2005 and December 2010. NHIRD is a comprehensive database of administrative and claim data which covers over 99% of entire population in Taiwan. Patients were selected with ICD codes and targeted therapy utilization, imatinib (IMA) or sunitinib (SUN). Time to treatment discontinuation (DC) due to any reasons (such as death, intolerance and disease progression) was estimated using Kaplan-Meier analysis. Results: From 2005 to 2010, the incidence rate of GIST in Taiwan was between 20 and 30 cases per million. A total of 3,436 GIST patients were identified; the mean age was 63.2 years and 57 % were male. Most patients (94.7%) received standard dose IMA (…400mg/day). Among these patients, 136 patients (4.0%) required IMA dose escalation and 44 (1.3%) patients switched to SUN after IMA. The probability of the 1st-line treatment DC was 20%, 30.7%, 38.9%, 43.2%, and 47.1% for years 1 to 5, respectively. The probability of a change in treatment pattern (defined as either IMA dose escalation or switch to SUN) was 4% 6.8%, 9.6%, 10.5% and 11.7% for years 1 to 5. Lastly, the probability of the 2nd-line treatment DC was 20.5%, 33.7%, 41.1%, 44.8%, 51.7% for years 1 to 5, respectively. Conclusions: First-line IMA plus other treatment modalities may provide additional benefits to progression-free survival. Taiwanese GIST patients who failed first-line treatment still gained benefit from either IMA dose escalation or a switch to SUN. Additional analysis is required for the detailed comparison in different treatment patterns.

Cost-effectiveness Analysis of Caspofungin and Fluconazole for Primary Treatment of Invasive Candidiasis and Candidemia in Ethiopia

2022 ◽  
Author(s):  
Gebremedhin Beedemariam Gebretekle ◽  
Atalay Mulu Fentie ◽  
Girma Tekle Gebremariam ◽  
Eskinder Eshetu Ali ◽  
Daniel Asfaw Erku ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Invasive Candidiasis ◽  
Cost Effective ◽  
Primary Treatment ◽  
Line Treatment ◽  
Second Line ◽  
First Line ◽  
System Perspective ◽  
The Cost ◽  
First Line Treatment

Abstract Background: Caspofungin was shown to be more effective than fluconazole in treating patients with invasive candidiasis and/or candidaemia (IC/C). However, cost-effectiveness of caspofungin for treating IC/C in Ethiopia remains unknown. We aimed to assess the cost-effectiveness of caspofungin compared to fluconazole as primary treatment of IC/C in Ethiopia.Methods: A Markov cohort model was developed to compare the cost-utility of caspofungin versus fluconazole antifungal agents as first-line treatment for adult inpatients with IC/C from the Ethiopian health system perspective. Treatment outcome was categorized as either a clinical success or failure, with clinical failure being switched to a different antifungal medication. Liposomal amphotericin B (L-AmB) was used as a rescue agent for patients who had failed caspofungin treatment, while caspofungin or L-AmB were used for patients who had failed fluconazole treatment. Primary outcomes were expected quality-adjusted life years (QALYs), costs (US$2021), and the incremental cost-effectiveness ratio (ICER). QALYs and costs were discounted at 3% annually. Cost data was obtained from Addis Ababa hospitals while locally unavailable data were derived from the literature. Cost-effectiveness was assessed against the recommended threshold of 50% of Ethiopia’s gross domestic product/capita. Deterministic and probabilistic sensitivity analyses were conducted to assess the robustness of the findings.Results: In the base-case analysis, treatment of IC/C with caspofungin as first-line treatment resulted in better health outcomes (12.86 QALYs) but higher costs (US$7,714) compared to fluconazole-initiated treatment followed by caspofungin (12.30 QALYs; US$3,217) or L-AmB (10.92 QALYs; US$2,781) as second-line treatment. Caspofungin as primary treatment for IC/C was not cost-effective when compared to fluconazole-initiated therapies. Fluconazole-initiated treatment followed by caspofungin was cost-effective for the treatment of IC/C compared to fluconazole with L-AmB as second-line treatment, at US$316/QALY gained. Our findings were sensitive to medication costs, drug effectiveness, infection recurrence, and infection-related mortality rates. Probabilistic sensitivity analysis confirmed the stability of our findings.Conclusions: Our study showed that the use of caspofungin as primary treatment for IC/C in Ethiopia was not cost-effective when compared with fluconazole-initiated treatment alternatives. The findings supported the use of fluconazole-initiated therapy with caspofungin as a second-line treatment to treat IC/C in Ethiopia and other low-income countries.

Outcome By Primary Treatment Type and Timing of Progression Among Follicular Lymphoma Patients: A Large, Population-Based Study in Sweden

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 46-47
Author(s):  
Caroline Weibull ◽  
Björn E Wahlin ◽  
Sandra Lockmer ◽  
Gunilla Enblad ◽  
Per-Ola Andersson ◽  
...  
Keyword(s):  
Research Funding ◽  
Primary Treatment ◽  
Population Based ◽  
Line Treatment ◽  
First Line ◽  
Population Based Study ◽  
Line Therapy ◽  
Progression Of Disease ◽  
First Line Treatment ◽  
Time Point

Purpose: Follicular lymphoma (FL) is generally regarded as an indolent malignancy, yet the clinical outcome is highly variable. In recent years, POD24 (progression of disease within 24 months) has emerged as a potential prognostic marker for overall survival (OS) in FL and other non-Hodgkin lymphomas. The association with survival, however, has mostly been studied in selected clinical trial cohorts and among patients treated with R-CHOP. We aimed to investigate OS by timing of progression and type of primary treatment in a population-based setting in Sweden. Methods: We identified all patients diagnosed with FL in stages II-IV and grade 1-3a between 2007 and 2014, using the population-based Swedish lymphoma register. Data were complemented with information on progression, transformation and second-line treatment through medical charts review up to December 31st, 2017. The analysis covered 4 out of 6 health care regions (75% of all patients diagnosed nationally). The patients were categorized according to type of first-line treatment: R-chemo of any type, R-Benda, R-CHOP (including R-CHOEP), or other (including immunotherapy only). Among patients where it was decided to start first-line treatment within 6 months of diagnosis (and where treatment was started within nine months), POD was defined as either lack of response to first-line therapy (stable [SD] or progressive disease [PD]), or initial response and subsequent relapse/progression/transformation as indication for second-line therapy. To quantify the impact of timing of POD on survival, the five-year OS conditional on either being progression-free (PF) or having experienced POD at different time points during follow-up, was estimated using a flexible parametric illness-death model. Results: Among a total of 970 FL patients, median age at diagnosis was 66 years and patients were followed for a median of 6.4 years (range 0-12 years). The 5-year OS was 75% and progression-free survival was 59%. Six hundred (62%) patients had a first-line treatment within nine months of diagnosis and were hence analyzed further, whereas the remaining 370 (38%) patients were classified as wait-and-watch and were not analyzed further. Among the 600 treated patients, 337 (56%) had R-chemo (R-CHOP or alike (n=210), R-Benda (n=97), other (n=30)), and 263 (44%) received non-R-chemo treatment (mainly R-monotherapy, radiotherapy only, or R-lenalidomide). Patients who received R-Benda were on average older than the other groups. Among patients treated with R-chemo, those who stayed progression-free had a 5-year conditional OS above 75% regardless of PF time point. For patients who progressed, the 5-year conditional OS improved as time point of POD was prolonged (Fig 1a, left panel). Early POD (within 12-24 months) was associated with a particularly poor prognosis (5-year conditional OS below 55%). The OS improvement over time of POD was especially pronounced among R-Benda treated patients (Fig 1b, right panel). Among patients receiving non-R-chemo treatments, early POD was associated with a slightly worse 5-year OS but differences between POD and PF patients were less marked (Fig 1a, right panel). Conclusion: This population-based study of Swedish stage II-IV FL patients shows that among immunochemotherapy-treated patients, progression of disease was always associated with worse survival in comparison to progression-free patients regardless of timing of progression. This reduction in survival was more pronounced the earlier the progression (as described by others). Interestingly, among patients selected for milder non-immunochemotherapy-based treatments, progression of disease did not have a strong effect on survival. Disclosures Weibull: Janssen Cilag: Research Funding. Wahlin:Gilead Sciences: Research Funding; Roche: Consultancy, Research Funding. Smedby:Takeda: Research Funding; Janssen: Research Funding; Celgene: Consultancy.

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