scholarly journals Tumor Reduction with Pazopanib in a Patient with Recurrent Lumbar Chordoma

2018 ◽  
Vol 2018 ◽  
pp. 1-7 ◽  
Author(s):  
Maurício Fernando Silva Almeida Ribeiro ◽  
Micelange Carvalho de Sousa ◽  
Samir Abdallah Hanna ◽  
Marcos Vinicius Calfat Maldaun ◽  
Ceci Obara Kurimori ◽  
...  
Keyword(s):  
Kinase Inhibitors ◽  
Systemic Treatment ◽  
Treatment Options ◽  
Axial Skeleton ◽  
Low Grade ◽  
Complete Excision ◽  
Systemic Treatments ◽  
Tumor Reduction ◽  
Comprehensive Genomic Profiling ◽  
Multifocal Recurrence

Introduction. Chordomas are rare malignancies of bone origin that occur in the axial skeleton, typically the skull base and lumbar/sacral regions. Although often classified as low-grade neoplasms, its locally infiltrative behavior may result in significant morbidity and mortality. Optimal surgical resection may be curative, but up to 50% of the cases relapse within 5 years, and currently there are no systemic treatments approved in this setting. A large proportion of these tumors express stem-cell factor receptor (c-KIT) and platelet-derived growth factor receptors (PDGFRs), providing a rationale for the use of tyrosine-kinase inhibitors (TKIs). Case report. A 27-year-old male presented with recurrent chordoma of the lumbar spine 4 years after initial diagnosis. Salvage therapies in the interval included repeat resections and radiation therapy. He ultimately developed multifocal recurrence not amenable to complete excision or reirradiation. A comprehensive genomic profiling assay was performed and revealed nondrugable alterations. Decision was made to proceed with systemic treatment with pazopanib 800 mg/day, resulting in tumor reduction (−23.1% reduction in size) and prolonged disease control. Conclusion. For this patient with a multiple recurrent chordoma and limited treatment options, pazopanib resulted in sustained clinical benefit following initial tumor reduction.

Management of carcinoid syndrome (CS): A systematic review of systemic treatment options.

2018 ◽  
Vol 36 (4_suppl) ◽  
pp. 516-516
Author(s):  
Edward M. Wolin ◽  
Al Bowen Benson
Keyword(s):  
Systematic Review ◽  
Clinical Trials ◽  
Systemic Treatment ◽  
Treatment Options ◽  
Symptom Relief ◽  
Safety Data ◽  
Significant Heterogeneity ◽  
Carcinoid Heart Disease ◽  
Systemic Treatments ◽  
Symptom Response

516 Background: CS is a major cause of symptom burden (diarrhea/flushing) in patients (pts) with neuroendocrine tumors and is associated with shortened survival and severe complications such as carcinoid heart disease. We performed a systematic review of clinical evidence for CS systemic treatments. Methods: PubMed, Embase, and Cochrane databases were searched. Large ( > 60 pt) prospective clinical trials investigating the efficacy/safety of systemic treatment options for pts with CS were eligible for inclusion. Data extracted included study design, pt population, interventions, prior treatments, permitted concomitant medications, study endpoints/statistical analyses, and efficacy/safety outcomes. Results: Six large prospective clinical trials (total 953 pts) were identified from 144 search results. Significant heterogeneity existed in pt populations, control groups (placebo vs active comparator), duration of therapy, study endpoints, and efficacy/safety data reporting. Interventions assessed were long-acting and subcutaneous octreotide (OCT & OCT SC), lanreotide (LAN), telotristat ethyl (TE)+somatostatin analogues (SSA), pasireotide (PAS), and everolimus (EVE)+OCT. Symptom response was variably assessed by the frequency of diarrhea/flushing and/or receipt of rescue short-acting SSA in 5/6 studies; a significant improvement in at least one of these measures occurred in 4 studies (OCT and OCT SC; LAN; TE+SSA). Failure to meet symptom response criteria ranged from 29-82% in the studies. Reported reductions in CgA and/or 5-HIAA reached statistical significance in 3 studies (LAN; EVE+OCT; TE+SSA). Interventions were generally well tolerated. Two phase 3 trials focused specifically on management of CS symptoms refractory to SSA; strategies included switching to PAS or an increased dose of OCT (40 mg q28d) for refractory diarrhea/flushing, or receipt of TE with continued SSA for refractory diarrhea. Conclusions: SSA provide substantial symptom relief for pts with CS. For refractory symptoms, TE can be added, SSA dose increased, and metastasis debulking can be used (with surgery, hepatic artery embolization, ablation, and PRRT). The addition of biologic agents to SSA can further improve CS management.

Bone and soft tissue malignancies

2015 ◽  
Author(s):  
Jim Cassidy ◽  
Donald Bissett ◽  
Roy A. J. Spence OBE ◽  
Miranda Payne ◽  
Gareth Morris-Stiff
Keyword(s):  
Myeloid Leukaemia ◽  
Hodgkin Lymphoma ◽  
Kinase Inhibitors ◽  
Treatment Options ◽  
B Cell Lymphoma ◽  
Current Treatment ◽  
Bisphosphonate Therapy ◽  
Heterogeneous Group ◽  
Low Grade ◽  
Quality Life

Haematological malignancies examines the epidemiology, genetics, clinical presentation and classification of these diseases, and presents current treatment approaches for each. First are the acute leukaemias, and the management of acute lymphoblastic leukaemia (ALL) and acute myeloid leukaemia (AML). Chronic myeloid leukaemia, its genetics and sensitivity to tyrosine kinase inhibitors, is described. Myelodysplastic syndromes and their management, are followed by chronic lymphoid leukaemias, a large heterogeneous group of diseases, and their treatment. Hodgkin lymphoma, its pathology and presentation, staging and role of PET scanning, is described along with current treatment with chemotherapy and limited radiotherapy. Non-Hodgkin lymphoma is another heterogeneous group of diseases, divided into low-grade and high-grade pathology, and varying in their genetics, presentation, and management. Rituximab is a key component of chemotherapy regimens against B-cell lymphoma. Myeloma and other plasma cell dyscrasias are described, and treatment options reviewed. Myeloma remains incurable, but with appropriate management consistent with prolonged good quality life. Treatment includes chemotherapy, bisphosphonate therapy, analgesics and radiotherapy, Throughout this chapter is emphasised the importance of clinical trials in driving the rapid improvements in treatment of these diseases.

A Treatment Algorithm for Moderate to Severe Atopic Dermatitis in Adults

2017 ◽  
Vol 22 (1) ◽  
pp. 78-83 ◽  
Author(s):  
Charles W. Lynde ◽  
Marc Bourcier ◽  
Melinda Gooderham ◽  
Lyn Guenther ◽  
Chih-ho Hong ◽  
...  
Keyword(s):  
Atopic Dermatitis ◽  
Systemic Treatment ◽  
Topical Therapy ◽  
Treatment Options ◽  
Treatment Algorithm ◽  
Current Evidence ◽  
Severe Atopic Dermatitis ◽  
Routine Practice ◽  
Clinical Criteria ◽  
Systemic Treatments

Background: Atopic dermatitis (AD) is a common and chronic inflammatory skin disease. Approximately 10% of adults with AD do not respond adequately to topical therapies and require phototherapy and/or systemic therapy. Objective: To provide a patient-focused approach to the identification and management of adults with AD who require systemic treatment. Methods: A working group of clinicians experienced in managing AD was convened to review and discuss current evidence on the identification and clinical management of adults with moderate to severe AD. Results: We propose a set of simple and practical clinical criteria for selecting candidates for systemic treatment of AD based on their response to first-line topical therapy and 4 clinical measures that are easily incorporated into routine practice. We also suggest a framework for evaluating systemic treatments according to attributes that are important from both a clinician’s and a patient’s perspective. An algorithm was developed proposing a pathway for treatment of moderate to severe AD in adults. Conclusion: Adults with moderate to severe AD that does not respond adequately to topical therapies currently have few safe and effective treatment options. A clinical algorithm could help guide treatment decisions.

scholarly journals Systemic Treatment in Advanced Phyllodes Tumor of the Breast: A Multi-institutional European Retrospective Case-series Analyses

2021 ◽  
Author(s):  
Elena Palassini ◽  
Olivier Mir ◽  
Giovanni Grignani ◽  
Bruno Vincenzi ◽  
Hanneke Gelderblom ◽  
...  
Keyword(s):  
Kinase Inhibitors ◽  
Systemic Treatment ◽  
Case Series ◽  
Phyllodes Tumor ◽  
Median Number ◽  
Advanced Breast ◽  
High Rate ◽  
High Dose ◽  
Retrospective Case ◽  
Systemic Treatments

Abstract Background: We aimed at investigating outcome of systemic treatments in advanced breast PT. Methods: All cases of advanced breast PT treated with systemic treatments from 1999 to 2019, in one of the referral sarcoma centres involved in the study, were retrospectively reviewed. Results: 56 female patients were identified. Median age was 52 (range 25-76) years. Patients re-ceived a median number of 2 systemic treatments (range 1-4). Best responses according to RECIST were: 1 (3.7%) CR, 11 (40.7%) PR, 6 (22.2%) SD, 9 (33.3%) PD with anthracyclines plus ifosfamide (AI); 2 (16.7%) PR, 4 (33.3%) SD, 6 (50.0%) PD with anthracycline alone; 3 (18.8%) PR, 4 (25.0%) SD, 9 (56.3%) PD with high-dose ifosfamide given as a continuous infusion (HD-IFX); 3 (20.0%) SD, 12 (80.0%) PD with a gemcitabine-based regimen (with 2 patients not evaluable); 1 (8.3%) PR, 2 (16.7%) SD, 9 (75.0%) PD with trabectedin (with 1 patient not evaluable); 1 (16.7%) PR, 1 (16.7%) SD, 4 (66.7%) PD with tyrosine-kinase inhibitors (TKI). The median PFS were: 5.7 (IQR, 2.5-9.1) months with AI; 3.2 (IQR, 2.2-5.0) months with anthracycline alone; 3.4 (IQR, 1.4-6.7) months with HD-IFX; 2.1 (IQR, 1.4-5.2) months with gemcitabine-based chemotherapy; 1.8 (IQR, 0.7-6.6) months with trabectedin; 3.4 (IQR, 3.1-3.8) months with TKI. With a median follow-up of 35.3 (IQR, 17.6-66.9) months, OS from the start of first-line systemic treatment was 15.2 (IQR, 7.6-39.6) months. Conclusion: In this series of advanced PT (to our knowledge, the largest reported so far), AI was associated with a high rate of responses, however with a median PFS of 5.7 months. Other systemic treatments were poorly active.

Employing RNA sequencing to enhance treatment options for cancer patients.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. 3628-3628
Author(s):  
Gargi D. Basu ◽  
Janine LoBello ◽  
Audrey Ozols
Keyword(s):  
Rna Sequencing ◽  
Myogenic Differentiation ◽  
Treatment Options ◽  
Targeted Treatment ◽  
Low Grade ◽  
Genomic Profiling ◽  
Molecular Abnormalities ◽  
Transcript Variants ◽  
Comprehensive Genomic Profiling ◽  
Variant Detection

3628 Background: Fusions and translocations account for 20% of cancer mortality globally. Maximizing their detection enhances the utility of precision medicine for various solid and hematologic cancers. Practice guidelines stress the importance of RNA sequencing. Novel assay techniques employing a comprehensive genomic profiling approach, including RNA sequencing, yield information beyond conventional DNA next generation sequencing (NGS) alone. Methods: Tumor samples (N = 1517) were assayed combining whole transcriptome (RNA) sequencing, whole exome (DNA) sequencing, and comparison of tumor sequence vs. paired normal DNA. Results were analyzed to determine the frequency of rare and common RNA fusion and variant detection. Findings were mapped to a knowledge-base of targeted treatment options. Results: Analysis detected 79 (5.2%) actionable fusions and 15 (1%) transcript variants across major solid and heme-based malignancies. Notably, we observed actionable transcript variants that are not detectable at the DNA level including: EGFRvIII, EGFRvIVa and EGFRvIVb in GBM; ARv7 in prostate, and METe14 in TNBC. Many fusion cases (42%, n = 33) had no other actionable molecular abnormalities. Novel fusions included: SLC12A/ROS1 in low-grade spindle cell neoplasm with myogenic differentiation, KANK1/NTRK2 in ganglioneuroblastoma, ETV6/NTRK3 in metastatic mammary analogue secretory carcinoma, FGFR1/SCT in germ cell tumor, ZNF33B/RET fusion in GBM, SH3BP4/ERBB4 and EML4/ALK in RCC, VTCN1/NRG1 in pancreatic cancer, and AGRN/NRG1 in cholangiocarcinoma. More common actionable fusion events included: EML4/ALK in NSCLC, KIAA1549/BRAF in pilocytic astrocytoma, FGFR2 and FGFR3 in cholangiocarcinoma and urothelial cancers and ESR1 in endocrine therapy-resistant breast cancers. The fusion events detected in heme-based malignancies included MLLT10 and MLLT4 in AML, BCR/ABL in leukemias, TCF3/PBX1 in B cell ALL, NPM1/ALK in ALCL, and novel fusion CIITA/CD274 in DLBCL. All RNA fusions and transcript variants found were matched to FDA-approved or investigational treatment options. Conclusions: Maximizing the rate of variant detection for targeted therapy relies on precise identification of common and rare fusion events. Without the addition of RNA sequencing, 15 transcript variants in our cohort would have been missed and 33 of the fusions may have gone undetected by conventional DNA NGS testing, resulting in zero targeted treatment options for this vulnerable population. Further use of comprehensive genomic profiling is vital to optimizing cancer care.

Review of Systemic Treatment Options for Adult Atopic Dermatitis

2016 ◽  
Vol 21 (1) ◽  
pp. 31-39 ◽  
Author(s):  
Melinda Gooderham ◽  
Charles W. Lynde ◽  
Kim Papp ◽  
Marc Bourcier ◽  
Lyn Guenther ◽  
...  
Keyword(s):  
Atopic Dermatitis ◽  
Immune Responses ◽  
Systemic Therapy ◽  
Systemic Treatment ◽  
Treatment Options ◽  
Skin Barrier ◽  
New Approach ◽  
Systemic Treatments ◽  
Systemic Therapies

Background: Atopic dermatitis (AD) is a chronic, pruritic inflammatory skin disease resulting from defects in skin barrier and aberrant immune responses. AD significantly affects the quality of life. Not all patients respond to topical therapies, and often systemic therapy is required to control the disease. Objective: To review the treatment options for adult AD patients including those options for patients who do not respond adequately or have contraindications to oral systemic therapy. Methods: A working group of clinicians with experience managing AD was convened to review the current literature on treatment options for adult AD patients. This review is based on the best available evidence from a published systematic review and an additional literature search. Results: Current treatments for AD are reviewed, including options for adult AD patients who do not respond or have contraindications to current systemic therapies. A new approach with targeted therapies is reviewed based on best available evidence. Conclusion: Many AD patients respond satisfactorily to topical or systemic treatments, but for those patients who do not respond or have contraindications, new biologic agents appear to be promising therapies.

scholarly journals Systemic Treatment in Advanced Phyllodes Tumor of the Breast: A Multi-Institutional European Retrospective Case-Series Analyses

2021 ◽  
Author(s):  
Elena Palassini ◽  
Olivier Mir ◽  
Giovanni Grignani ◽  
Bruno Vincenzi ◽  
Hans Gelderblom ◽  
...  
Keyword(s):  
Kinase Inhibitors ◽  
Systemic Treatment ◽  
Case Series ◽  
Phyllodes Tumor ◽  
Median Number ◽  
Advanced Breast ◽  
High Rate ◽  
High Dose ◽  
Retrospective Case ◽  
Systemic Treatments

Abstract Background: We aimed at investigating outcome of systemic treatments in advanced breast PT. Methods: All cases of advanced breast PT treated with systemic treatments from 1999 to 2019, in one of the referral sarcoma centres involved in the study, were retrospectively reviewed. Results: 56 female patients were identified. Median age was 52 (range 25-76) years. Patients re-ceived a median number of 2 systemic treatments (range 1-4). Best responses according to RECIST were: 1 (3.7%) CR, 11 (40.7%) PR, 6 (22.2%) SD, 9 (33.3%) PD with anthracyclines plus ifosfamide (AI); 2 (16.7%) PR, 4 (33.3%) SD, 6 (50.0%) PD with anthracycline alone; 3 (18.8%) PR, 4 (25.0%) SD, 9 (56.3%) PD with high-dose ifosfamide given as a continuous infusion (HD-IFX); 3 (20.0%) SD, 12 (80.0%) PD with a gemcitabine-based regimen (with 2 patients not evaluable); 1 (8.3%) PR, 2 (16.7%) SD, 9 (75.0%) PD with trabectedin (with 1 patient not evaluable); 1 (16.7%) PR, 1 (16.7%) SD, 4 (66.7%) PD with tyrosine-kinase inhibitors (TKI). The median PFS were: 5.7 (IQR, 2.5-9.1) months with AI; 3.2 (IQR, 2.2-5.0) months with anthracycline alone; 3.4 (IQR, 1.4-6.7) months with HD-IFX; 2.1 (IQR, 1.4-5.2) months with gemcitabine-based chemotherapy; 1.8 (IQR, 0.7-6.6) months with trabectedin; 3.4 (IQR, 3.1-3.8) months with TKI. With a median follow-up of 35.3 (IQR, 17.6-66.9) months, OS from the start of first-line systemic treatment was 15.2 (IQR, 7.6-39.6) months. Conclusion: In this series of advanced PT (to our knowledge, the largest reported so far), AI was associated with a high rate of responses, however with a median PFS of 5.7 months. Other systemic treatments were poorly active.

scholarly journals Limited Liver or Lung Colorectal Cancer Metastases. Systemic Treatment, Surgery, Ablation or SBRT

2021 ◽  
Vol 10 (10) ◽  
pp. 2131
Author(s):  
Meritxell Molla ◽  
Julen Fernandez-Plana ◽  
Santiago Albiol ◽  
Constantino Fondevila ◽  
Ivan Vollmer ◽  
...  
Keyword(s):  
Colorectal Cancer ◽  
Systemic Treatment ◽  
Lung Metastases ◽  
Treatment Options ◽  
Percutaneous Ablation ◽  
Multidisciplinary Teams ◽  
Cancer Metastases ◽  
Systemic Treatments ◽  
Challenges And Opportunities ◽  
Treatment Surgery

The prognosis for oligometastatic colorectal cancer has improved in recent years, mostly because of recent advances in new techniques and approaches to the treatment of oligometastases, including new surgical procedures, better systemic treatments, percutaneous ablation, and stereotactic body radiation therapy (SBRT). There are several factors to consider when deciding on the better approach for each patient: tumor factors (metachronous or synchronous metastases, RAS mutation, BRAF mutation, disease-free interval, size and number of metastases), patient factors (age, frailty, comorbidities, patient preferences), and physicians’ factors (local expertise). These advances have presented major challenges and opportunities for oncologic multidisciplinary teams to treat patients with limited liver and lung metastases from colorectal cancer with a curative intention. In this review, we describe the different treatment options in patients with limited liver and lung metastases from colorectal cancer, and the possible combination of three approaches: systemic treatment, surgery, and local ablative treatments.

Immunotherapy and potential predictive biomarkers in the treatment of neuroendocrine neoplasia

2020 ◽  
Author(s):  
Guanghui Xu ◽  
Yuhao Wang ◽  
Hushan Zhang ◽  
Xueke She ◽  
Jianjun Yang
Keyword(s):  
Current Knowledge ◽  
Checkpoint Inhibitors ◽  
Treatment Options ◽  
Predictive Biomarkers ◽  
The Body ◽  
Low Grade ◽  
Ablative Therapies ◽  
Cancer Types ◽  
New Treatment ◽  
Well Differentiated

Neuroendocrine neoplasias (NENs) are a heterogeneous group of rare tumors scattered throughout the body. Surgery, locoregional or ablative therapies as well as maintenance treatments are applied in well-differentiated, low-grade NENs, whereas cytotoxic chemotherapy is usually applied in high-grade neuroendocrine carcinomas. However, treatment options for patients with advanced or metastatic NENs are limited. Immunotherapy has provided new treatment approaches for many cancer types, including neuroendocrine tumors, but predictive biomarkers of immune checkpoint inhibitors (ICIs) in the treatment of NENs have not been fully reported. By reviewing the literature and international congress abstracts, we summarize the current knowledge of ICIs, potential predicative biomarkers in the treatment of NENs, implications and efficacy of ICIs as well as biomarkers for NENs of gastroenteropancreatic system, lung NENs and Merkel cell carcinoma in clinical practice.

scholarly journals Characteristics and Treatment Challenges of Non-Clear Cell Renal Cell Carcinoma

Cancers ◽  
2021 ◽  
Vol 13 (15) ◽  
pp. 3807
Author(s):  
Pierangela Sepe ◽  
Arianna Ottini ◽  
Chiara Carlotta Pircher ◽  
Andrea Franza ◽  
Melanie Claps ◽  
...  
Keyword(s):  
Renal Cell ◽  
Kinase Inhibitors ◽  
Clear Cell ◽  
Case Reports ◽  
Treatment Options ◽  
Single Agent ◽  
Growth Factor Receptor ◽  
Mtor Inhibitors ◽  
Cell Renal Cell Carcinoma ◽  
Clear Cell Rcc

Non-clear cell renal cell carcinomas (RCC) comprise several rare and poorly described diseases, often characterized by bad prognosis and with no standard treatments available. The gap in their clinical management is linked to the poor molecular characterization in handling the treatment of non clear-cell RCC with untailored therapies. Due to their rarity, non-clear RCC are in fact under-represented in prospective randomized trials. Thus, treatment choices are based on extrapolating results from clear cell RCC trials, retrospective data, or case reports. Over the last two decades, various options have been considered as the mainstay for the treatment of metastatic RCC (mRCC), including angiogenesis inhibitors, vascular endothelial growth factor receptor inhibitors, other tyrosine kinase inhibitors (TKIs), as well as MET inhibitors and mammalian targeting of rapamycin (mTOR) inhibitors. More recently, the therapeutic armamentarium has been enriched with immunotherapy, alone or in combination with targeted agents that have been shown to significantly improve outcomes of mRCC patients, if compared to TKI single-agent. It has been widely proven that non-clear cell RCC is a morphologically and clinically distinct entity from its clear cell counterpart but more knowledge about its biology is certainly needed. Histology-specific collaborative trials are in fact now emerging to investigate different treatments for non-clear cell RCC. This review summarizes pathogenetic mechanisms of non-clear cell RCC, the evolution of treatment paradigms over the last few decades, with a focus on immunotherapy-based trials, and future potential treatment options.

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