Ipertiroidismo e tiroidite autoimmune in età evolutiva

2021 ◽  
Vol 40 (10) ◽  
pp. 637-645
Author(s):  
Maria Chiara Pellegrin ◽  
Alessandro Occhipinti ◽  
Benedetta Bossini ◽  
Stefania Norbedo ◽  
Elena faleschini ◽  
...  
Keyword(s):  
Remission Rate ◽  
Treatment Options ◽  
Spontaneous Remission ◽  
Antithyroid Drugs ◽  
First Line ◽  
First Line Therapy ◽  
Line Therapy ◽  
Antithyroid Treatment ◽  
Paediatric Age ◽  
Optimal Regimen

Hyperthyroidism is a rare clinical entity in paediatric age. In most cases the etiology is autoimmune and goiter represents the typical presentation. Ophthalmopathy is rare and can precede the diagnosis of autoimmune hyperthyroidism. Thyrotoxicosis caused by the hyperthyroid phase of Hashimoto’s thyroiditis must be carefully distinguished from Graves’ disease (GD), since the first condition has a better prognosis for spontaneous remission. Three treatment options are currently available for the management of paediatric GD. First-line therapy is antithyroid drugs, while thyroidectomy and radioiodine are considered on relapse. A lower remission rate and a higher risk of adverse events are observed after the first course of methimazole in childhood with respect to adulthood. Children and adolescents may require a prolonged antithyroid treatment, but an overall consensus regarding the optimal regimen is lacking.

Phase 2 study of pembrolizumab as first-line therapy for PD-L1–positive metastatic triple-negative breast cancer (mTNBC): Preliminary data from KEYNOTE-086 cohort B.

2017 ◽  
Vol 35 (15_suppl) ◽  
pp. 1088-1088 ◽  
Author(s):  
Sylvia Adams ◽  
Sherene Loi ◽  
Deborah Toppmeyer ◽  
David W. Cescon ◽  
Michele De Laurentiis ◽  
...  
Keyword(s):  
Antitumor Activity ◽  
Tumor Imaging ◽  
Treatment Options ◽  
First Line ◽  
First Line Therapy ◽  
Line Therapy ◽  
New Treatment ◽  
Combined Positive Score ◽  
Ecog Ps

1088 Background: Standard first-line treatment for mTNBC is chemotherapy. However, outcomes are poor, and new treatment options are needed. Cohort B of KEYNOTE-086 (NCT02447003) assessed the safety and antitumor activity of pembrolizumab as first-line therapy for patients (pts) with PD-L1–positive mTNBC. Methods: Men and women with centrally confirmed mTNBC, no prior systemic anticancer therapy for metastatic disease, ECOG PS 0-1, and a tumor PD-L1 combined positive score (CPS) ≥1% received pembrolizumab 200 mg Q3W for 24 mo or until disease progression, intolerable toxicity, or investigator or pt decision. Tumor imaging was performed Q9W for 12 mo and Q12W thereafter. Clinically stable pts with PD could remain on pembrolizumab until PD was confirmed on subsequent assessment. Primary end point was safety. Secondary end points included ORR, DOR, and PFS (RECIST v1.1, central review). Planned enrollment was 80 pts. This analysis included all pts who had ≥18 wk of follow-up as of Nov 10, 2016. Results: 79 of the first 137 pts with PD-L1–evaluable tumors (58%) had PD-L1 CPS ≥1%. Of the first 52 pts enrolled, 100% were women, median age was 53 y, 40% had elevated LDH, 69% had visceral metastases, and 87% received prior (neo)adjuvant therapy. After a median follow-up of 7.0 mo (range 4.4-12.5), 15 (29%) pts remained on pembrolizumab. Treatment-related AEs occurred in 37 (71%) pts, most commonly fatigue (31%), nausea (15%), and diarrhea (13%). 4 (8%) pts experienced 5 grade 3-4 treatment-related AEs: back pain, fatigue, hyponatremia, hypotension, and migraine (n = 1 each). No pts died or discontinued pembrolizumab due to an AE. ORR was 23% (95% CI 14%-36%). Best overall response was CR in 4%, PR in 19%, SD in 17%, PD in 58%, and not assessed in 2%. Median time to response was 8.7 wk (range 8.1-17.7). Median DOR was 8.4 mo (range, 2.1+ to 8.4), with 8 (67%) responses ongoing at cutoff. Median PFS was 2.1 mo (95% CI, 2.0-3.9); estimated 6-mo PFS rate was 29%. Conclusions: Data from the first 52 pts enrolled in KEYNOTE-086 cohort B suggest that pembrolizumab monotherapy has a manageable safety profile and promising antitumor activity as first-line therapy for PD-L1–positive mTNBC. Clinical trial information: NCT02447003.

A Retrospective Population-Based Study of a Series of 307 Treatment-naïve Patients with Chronic Lymphocytic Leukemia (CLL): Study of the Clinical Features and Efficacy of First-Line Therapy

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 2452-2452
Author(s):  
Eva Ma Donato ◽  
Rafa Andreu ◽  
Aurelio Lopez ◽  
Ana Vicente ◽  
Ana Carral ◽  
...  
Keyword(s):  
Lymphocyte Count ◽  
Response Rate ◽  
Treatment Options ◽  
Line Treatment ◽  
First Line ◽  
Community Based ◽  
First Line Therapy ◽  
Cd38 Expression ◽  
Line Therapy ◽  
Purine Analogs

Abstract Abstract 2452 Background. Clinical trials have shown an improved response rate and progression free survival (PFS) among the different treatment options used in the last two decades, specially with rituximab in combination with fludarabine and cyclophosphamide. We wished to analyze, in an unselected community based population, the clinical characteristics and efficacy of first line therapy with several treatment options used throughout a ten-year period. Patients and methods. We included 307 patients diagnosed of CLL and requiring first-line treatment between January 2000 and September 2009. Patients were treated at 20 hospitals placed in the Community of Valencia and Murcia and received first-line therapy according to the clinical guidelines of each hospital. PFS was calculated from date of first treatment to date of progression/relapse. We performed a descriptive analysis of clinical and biological features. The survival curves were built with Kaplan-Meier method and compared with the log rank test. Multivariate analysis for response and PFS were performed by logistic and Cox regression methods respectively, using the statistical package SPSS (v15). Results. Median age at treatment was 67 years (range 28–94) with 58% (n=179) men. 39% (120/305) were in Binet A, 38% (117) in Binet B and 22% (68) in Binet C. 27% (84) were Rai III-IV. B symptoms were present in 25% (77) and fever was a rare symptom 3%. Patients were asymptomatic in 59% (181) of the cases with ECOG performance status 0–1 in 83% (256). Splenomegaly was present in 41% (127) and hepatomegaly only in 8% (24). 42% of the patients (129) had at least three lymph-node areas affected with bulky disease (diameter higher than >5cm) in 10% (32). Median haemoglobin level was 126gr/L (46–169), lymphocyte count 49 x109/L (0,5–613) with lymphocyte doubling time (LDT) <12 months in 43%. LDH was elevated in 34% (95/284) and b2-microglobulin levels in 61% (154/251). CD38 expression was positive in 42% (107/255) and ZAP-70 in 75% (91/122). The overall incidence of trysomy 12, 13q, 11q23 and 17p deletions detected by FISH were 17%, 15%, 12% and 5% respectively. Median follow-up was 33.5 months (0.6–156). First line treatment schedules and treatment response in every group are detailed in table 1. Patients receiving rituximab (group 3–4, n=73) achieved a significant higher response rate (CR or PR) than patients without rituximab (93% vs 61%). The main clinical variables with prognosis significance in the univariate analysis for PFS were: lymphocyte count (p=0,026, HR 1.002, CI95% (1.000–1.004)); haemoglobin level (p=0,02, HR 0.890, CI95% (0.826–0.958); b2-microglobulin (p=0,002, HR 1.372, CI95% (1.125–1.672)); bulky mass (p=0,055, HR 1.656, CI95% (0.989–2.775)); CD38 expression (p=0,045, HR 1.005, CI95% (1.000–1.011)); p53 deletion (p=0,014, HR 2,231, CI95% (1,180–4,217)) and 11q23 deletion (p=0,03, HR 2,138, CI95% (1,290–3,542)). The median PFS for patients in the different groups were: G1:26.9 months (22.4–31.3), G2 45.5 months (32.7–58.4), G3: not reached, G4: 20.5 months (29.6–47.3), p < 0.0001. In the multivariate analysis the variables with independent prognostic significance for PFS were: lymphocyte count (p=0.003, HR 1.004, CI 95% (1.001 −1.007)), 17deletion (p=0.01, HR 2.7 CI95% (1.273–5.73)), 11q deletion (p=0.006, HR 2.193 CI95% (1.248–3.852)) and treatment received (G1 as reference): G2 (p=0.005, HR 0.464, CI95% (0.271–0.794)), G3 (p < 0.001, HR 0.179, CI95% (0.083–0.386)), G4 (p=0.223, HR 0.289, CI95% (0.039–2.130)). Conclusion. In this community based population, treatment with rituximab containing regimens results in a higher global and complete response rate and a longer PFS compared with alquilating agents and purine analogs. Fludarabine containing-schedules also achieved a significant higher response rate than alquilating agents. Rituximab in combination with purine analogs provide the better quality of response. These results confirm the data provided by clinical trials and support their use as front-line treatment. Disclosures: Terol: ROCHE: Consultancy.

scholarly journals Immune Checkpoint Inhibitors for Unresectable Hepatocellular Carcinoma

Vaccines ◽  
2020 ◽  
Vol 8 (4) ◽  
pp. 616
Author(s):  
Mohamed A. Abd El Aziz ◽  
Antonio Facciorusso ◽  
Tarek Nayfeh ◽  
Samer Saadi ◽  
Mohamed Elnaggar ◽  
...  
Keyword(s):  
Hepatocellular Carcinoma ◽  
Immune Checkpoint ◽  
Immune Checkpoint Inhibitors ◽  
Checkpoint Inhibitors ◽  
Treatment Options ◽  
Survival Outcomes ◽  
Efficacy And Safety ◽  
First Line ◽  
First Line Therapy ◽  
Line Therapy

Despite the advances in screening protocols and treatment options, hepatocellular carcinoma (HCC) is still considered to be the most lethal malignancy in patients with liver cirrhosis. Moreover, the survival outcomes after failure of first-line therapy for unresectable HCC is still poor with limited therapeutic options. One of these options is immune checkpoint inhibitors. The aim of this study is to comprehensively review the efficacy and safety of immune checkpoint inhibitors for patients with HCC.

scholarly journals Will biological agents supplant systemic glucocorticoids as the first-line treatment for thyroid-associated ophthalmopathy?

2019 ◽  
Vol 181 (5) ◽  
pp. D27-D43 ◽  
Author(s):  
Terry J Smith ◽  
Luigi Bartalena
Keyword(s):  
Thyroid Autoimmunity ◽  
Treatment Options ◽  
Biological Agents ◽  
First Line ◽  
First Line Therapy ◽  
Line Therapy ◽  
Thyroid Associated Ophthalmopathy ◽  
Systemic Glucocorticoids ◽  
Points Of View ◽  
First Line Treatment

In this article, the two authors present their opposing points of view concerning the likelihood that glucocorticoids will be replaced by newly developed biological agents in the treatment of active, moderate-to-severe thyroid-associated ophthalmopathy (TAO). TAO is a vexing, disfiguring and potentially blinding autoimmune manifestation of thyroid autoimmunity. One author expresses the opinion that steroids are nonspecific, frequently fail to improve the disease and can cause sometimes serious side effects. He suggests that glucocorticoids should be replaced as soon as possible by more specific and safer drugs, once they become available. The most promising of these are biological agents. The other author argues that glucocorticoids are proven effective and are unlikely to be replaced by biologicals. He reasons that while they may not uniformly result in optimal benefit, they have been proven effective in many reports. He remains open minded about alternative therapies such as biologicals but remains skeptical that they will replace steroids as the first-line therapy for active, moderate-to-severe TAO without head-to-head comparative clinical trials demonstrating superiority. Despite these very different points of view, both authors are optimistic about the availability of improved medical therapies for TAO, either as single agents or in combination. Further, both agree that better treatment options are needed to improve the care of our patients with active moderate-to-severe TAO.

scholarly journals Peculiarities of topical therapy in pediatric psoriasis

2021 ◽  
Vol 16 (S4) ◽  
pp. 54-57
Author(s):  
Ana Maria Alexandra STĂNESCU ◽  
◽  
Vlad DIMA ◽  
Ioana Veronica GRĂJDEANU ◽  
Mira FLOREA ◽  
...  
Keyword(s):  
Children And Adolescents ◽  
Topical Therapy ◽  
Treatment Options ◽  
Efficacy And Safety ◽  
First Line ◽  
First Line Therapy ◽  
Line Therapy ◽  
Atypical Manifestations ◽  
Chromosomal Loci ◽  
Therapy Treatment

In children and adolescents, psoriasis affects between 0.5% and 1.2% of the population. Currently, at least 80 chromosomal loci have been discovered that are significantly linked to psoriasis, and some of these loci are mainly linked to the onset of psoriasis in childhood. Pediatric psoriasis can be more difficult to diagnose due to an easier course of the disease, with smaller and thinner plaques, which are less scalable and with atypical manifestations. First-line therapy in psoriasis in both children and adults is represented by topical therapy. Treatment options for psoriasis in children and adolescents are largely off-label, with little data available on efficacy and safety.

scholarly journals Systematic Literature Review on Global Real-World Treatment Patterns of Mantle Cell Lymphoma (MCL)

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 5881-5881
Author(s):  
Keri Keri Yang ◽  
Beth Lesher ◽  
Eleanor Lucas ◽  
Tony Caver ◽  
Boxiong Tang
Keyword(s):  
Literature Review ◽  
Adverse Events ◽  
Real World ◽  
Treatment Options ◽  
Treatment Patterns ◽  
Second Line ◽  
First Line ◽  
First Line Therapy ◽  
Line Therapy ◽  
Third Line

Introduction: MCL is an aggressive type of non-Hodgkin's lymphoma, and was reported associated with early relapse and poor long-term survival. Treatment options include chemotherapy, immunotherapy, and molecular targeted therapies. As of 2019, molecular targeted therapies available in the United States indicated for the treatment of MCL include the proteasome inhibitor bortezomib, the immunomodulatory drug lenalidomide (following two previous lines of therapy), and the Burton's tyrosine kinase inhibitors (BTKIs) ibrutinib and acalabrutinib (following at least one previous line of therapy). Objective/Methods: To examine the real-world treatment patterns of patients with MCL globally, a systematic literature review was performed (2010-2019) with predefined methodology and inclusion and exclusion criteria. Embase and Medline were searched via ProQuest and the Cochrane Controlled Register of Trials (CENTRAL) via the Cochrane Library. Results: Of the 2207 publications identified, 6 publications (US, n = 4; EU, n = 2) provided information on the first-line treatment of MCL (Table). The most commonly administered first-line treatments were bendamustine-rituximab; high dose cytarabine ± rituximab; and cyclophosphamide, doxorubicin hydrochloride, vincristine sulfate, and prednisone (CHOP) ± rituximab although differences were noted across studies. Most patients received rituximab first-line either in combination with chemotherapy (54.2%-87.5%) or as monotherapy (12.9%-28.9%); although in some studies, rituximab maintenance therapy could not be excluded. The most commonly administered second-line therapies were cytarabine, rituximab monotherapy, and ibrutinib while third-line therapies were rituximab monotherapy, ibrutinib, and temsirolimus. Nine studies provided data on the real-world treatment of MCL with the BTKI ibrutinib (EU, n = 3; US, n = 5; EU/US, n = 1; Table); no real-world studies were identified for acalabrutinib. Six studies enrolled patients only with relapsed or remitting MCL; 3 studies enrolled patients (≤7.5%) who received ibrutinib as first-line therapy. Ibrutinib second-line therapy was administered to 13%-20% of patients and third-line therapy to 21% of patients. Ibrutinib discontinuation rates in 7 studies varied from 38.7%-83.6%. Non-response, including relapse or progression (34.6%-100%), was the main cause of discontinuation, followed by toxicity/adverse events (8.1%-25.6%). Across studies, toxicity/adverse events causing ibrutinib discontinuation included atrial fibrillation, bleeding/hemorrhage, chronic obstructive pulmonary disease, diarrhea, herpes zoster, infection, leukocytosis/ lymphocytosis, lung cancer, myelodysplastic syndrome, and thrombocytopenia. Two studies provided information on ibrutinib dose reductions (16.4% of patients) and ibrutinib dose interruptions (7.8%-30.2% of patients). Treatment options administered post-ibrutinib included rituximab (52.7%), hyper-CVAD + rituximab (16.7%-25.8%), lenalidomide-based regimens (9.7%-41.5%), and bortezomib-based regimens (8.4%-34.4%). Conclusion: Our analyses showed that most patients with MCL received first-line chemoimmunotherapy, although regimens varied across studies. Approximately 13%-21% of patients received ibrutinib following first-line therapy. Most ibrutinib discontinuation was due to progression followed by toxicity/adverse events. Upon discontinuation of ibrutinib, considerable variation in treatments was seen and no standard therapy identified. Given the limitations of current therapies, there is a need for additional second- and third-line treatments for patients with MCL. Quantitative assessments of clinical endpoints from real-world studies evaluating BTKI therapies are also warranted. Disclosures Yang: BeiGene, Ltd.: Employment. Lesher:Pharmerit: Employment. Lucas:Pharmerit: Employment. Caver:BeiGene, Ltd.: Employment. Tang:BeiGene, Ltd.: Employment.

First-line therapy with fludarabine compared with chlorambucil does not result in a major benefit for elderly patients with advanced chronic lymphocytic leukemia

Blood ◽  
2009 ◽  
Vol 114 (16) ◽  
pp. 3382-3391 ◽  
Author(s):  
Barbara F. Eichhorst ◽  
Raymonde Busch ◽  
Stephan Stilgenbauer ◽  
Martina Stauch ◽  
Manuela A. Bergmann ◽  
...  
Keyword(s):  
Chronic Lymphocytic Leukemia ◽  
Elderly Patients ◽  
Survival Time ◽  
Remission Rate ◽  
Progression Free Survival ◽  
Lymphocytic Leukemia ◽  
Phase Iii ◽  
First Line ◽  
First Line Therapy ◽  
Line Therapy

Abstract Although chronic lymphocytic leukemia (CLL) is a disease of elderly patients, subjects older than 65 years are heavily underrepresented in clinical trials. The German CLL study group (GCLLSG) initiated a multicenter phase III trial for CLL patients older than 65 years comparing first-line therapy with fludarabine with chlorambucil. A total of 193 patients with a median age of 70 years were randomized to receive fludarabine (25 mg/m2 for 5 days intravenously, every 28 days, for 6 courses) or chlorambucil (0.4 mg/kg body weight [BW] with an increase to 0.8 mg/kg, every 15 days, for 12 months). Fludarabine resulted in a significantly higher overall and complete remission rate (72% vs 51%, P = .003; 7% vs 0%, P = .011). Time to treatment failure was significantly shorter in the chlorambucil arm (11 vs 18 months; P = .004), but no difference in progression-free survival time was observed (19 months with fludarabine, 18 months with chlorambucil; P = .7). Moreover, fludarabine did not increase the overall survival time (46 months in the fludarabine vs 64 months in the chlorambucil arm; P = .15). Taken together, the results suggest that in elderly CLL patients the first-line therapy with fludarabine alone does not result in a major clinical benefit compared with chlorambucil. This trial is registered with www.isrctn.org under identifier ISRCTN 36294212.

scholarly journals Epidemiological Analysis and Geographical Distribution of Patients Suffering from Rheumatoid Arthritis, Eligible for First-Line Therapy/Monotherapy Treatment with Subcutaneous Formulation of Tocilizumab in the Republic of Macedonia

PRILOZI ◽  
2018 ◽  
Vol 39 (2-3) ◽  
pp. 69-78
Author(s):  
Filip Gucev ◽  
Snezana Perchinkova-Mishevska ◽  
Georgi Bozinovski ◽  
Irena Kafedjiska ◽  
Dubravka Antova ◽  
...  
Keyword(s):  
Rheumatoid Arthritis ◽  
Treatment Option ◽  
Structural Damage ◽  
Treatment Options ◽  
Personal Data ◽  
Precise Determination ◽  
First Line ◽  
First Line Therapy ◽  
Line Therapy ◽  
Number Of Patients

Abstract Rheumatoid arthritis is an inflammatory arthritis characterized by synovial tissue inflammation that leads to structural damage and disability. There are several treatment options available, which include glucocorticoids, DMARDs and biologics given alone as monotherapy or in a variety of combinations. Recent evidence has shown that early treatment is important in reducing the rate of progression of erosions and decreasing disability. The lack of adequate statistical data on number of patients that are eligible for first-line therapy/monotherapy of rheumatoid arthritis in Macedonia, triggered this epidemiological analyse describing eligible patients for first-line treatment/monotherapy distributed by gender, age and geographical allocation. The study was conducted by fulfilling a tailored questionnaire every two months in a period of six months (September 2017-February 2018) by including summarized data not related to personal data of patients nor specific drug information. The results have shown that a total of 115 patients in Macedonia are eligible for first-line therapy, whereby 54 (46%) patients were eligible for monotherapy of rheumatoid arthritis. Precise determination of these data provides patients’ determination by geographical allocation and proper selection of the best treatment option and optimized therapy for each patient, furthermore when subcutaneous formulation of tocilizumab is available as an effective clinically proven treatment option for RA

scholarly journals Clinicopathological characteristics, diagnosis and treatment of melanoma in Serbia: The Melanoma Focus Study

2015 ◽  
Vol 72 (4) ◽  
pp. 312-316 ◽  
Author(s):  
Lidija Kandolf-Sekulovic ◽  
Nada Babovic ◽  
Mirjana Balic ◽  
Borislava Nikolin ◽  
Dejan Nikolic ◽  
...  
Keyword(s):  
Metastatic Melanoma ◽  
Clinical Studies ◽  
Treatment Options ◽  
Stage Iv ◽  
Second Line ◽  
First Line ◽  
Stage Iiic ◽  
First Line Therapy ◽  
Line Therapy ◽  
Melanoma Patients

Background/Aim. Treatment options for metastatic melanoma in Serbia are limited due to the lack of newly approved biologic agents and the lack of clinical studies. Also, there is a paucity of data regarding the treatment approaches in different tertiary centers and efficacy of available chemotherapy protocols. The aim of this study was to obtain more detailed data about treatment protocols in Serbia based on structured survey in tertiary oncology centers. Methods. Data about the melanoma patients treated in 2011 were analyzed from hospital databases in 6 referent oncology centers in Serbia, based on the structured survey, with the focus on metastatic melanoma patients (unresectable stage IIIC and IV). Results. A total of 986 (79-315 in different centers) patients were treated, with 320 (32.45%) newly diagnosed patients. There were 317 patients in stage IIIC/IV, 77/317 aged < 50 years. At the time of diagnosis 47.3% of patients were < 60 years of age (24.2% < 40 years, 23% 50-59 years, 52.6% > 60 years). At initial diagnosis 12.5% of patients were in stage III and 4.5% in stage IV. The most common type was superficial spreading melanoma (50-66%), followed by nodular melanoma (23.5-50%). Apart from the regional and distant lymph node metastases, the most frequent organs involved in stage IV disease were distant skin and soft tissues (12-55%), lungs (19-55.5%), liver (10-60%), and bones (3-10%). The first line therapy in stage IV metastatic melanoma was dacarbazine (DTIC) dimethyl-triazeno-imidozole-carboxamide in 61-93% of the patients, while the second line varied between the centers. Disease control (complete response + partial response + stable disease) was achieved in 25.7% of the patients treated with the first line chemotherapy and 23.1% of the patients treated with the second line therapy, but the duration of response was short, in first-line therapy 6.66 ? 3.36 months (median 6.75 months). More than 90% of patients were treated outside the clinical trials. Conclusion. Based on this survey, there is a large unmet need for the new treatment options for metastatic melanoma in Serbia. The development of national guidelines, and greater involvement in international clinical studies could lead to widening of treatment options for this chemotherapy resistant disease.

Sign in / Sign up

Export Citation Format

Share Document