Anti-IL1 treatment in colchicine-resistant paediatric FMF patients: real life data from the HELIOS registry

Rheumatology ◽  
2020 ◽  
Vol 59 (11) ◽  
pp. 3324-3329 ◽  
Author(s):  
Erdal Sag ◽  
Fuat Akal ◽  
Erdal Atalay ◽  
Ummusen Kaya Akca ◽  
Selcan Demir ◽  
...  
Keyword(s):  
Age At Onset ◽  
Real Life ◽  
Treated Group ◽  
First Line ◽  
Skin Reactions ◽  
Web Based ◽  
Local Skin ◽  
On Demand ◽  
Real Life Data ◽  
Drug Registry

Abstract Objectives FMF is a prototype of autoinflammatory diseases associated with excess IL1 production. Anti-IL1 treatments are the first-line alternatives in colchicine-resistant/intolerant FMF patients. We aimed to investigate the efficacy and safety of anti-IL1 treatment in paediatric FMF patients in our local [Hacettepe univErsity eLectronIc research fOrmS (HELIOS)] registry. Methods HELIOS is a web-based biologic drug registry for paediatric rheumatology patients. We have analysed the clinical features, disease activity parameters, treatment responses and safety outcomes in FMF patients treated with anti-IL1 agents. Results Forty paediatric FMF patients (34 continuous and six on-demand use) were included. Among the continuously treated group (61.7% female), the mean age at the start of colchicine was 5.55 (3.87) years. Age at onset of the anti-IL1 treatment was 11.47 (5.41) years with a mean follow-up duration of 3.87 (1.96) years. Apart from two, all patients had biallelic exon-10 mutations. We also gave anti-IL1 treatment on an on-demand basis in six patients. Anakinra was used as the first-line anti-IL1 treatment. During the last visit, six patients were treated with anakinra and 28 patients with canakinumab. Anti-IL1 treatment decreased the CRP levels and number and severity of the attacks. There were three hospitalizations reported due to mild infections. Eleven patients had local skin reactions, two patients had leucopenia with anakinra and one patient had thrombocytopenia with canakinumab. There was no malignancy or other severe adverse reactions. Conclusion Anakinra and canakinumab are efficient and safe alternatives in colchicine-resistant or -intolerant paediatric FMF patients. We also, for the first time, report on-demand use of anti-IL1 in paediatric FMF patients.

scholarly journals OP0288 ANTI-IL1 TREATMENT IN COLCHICINE RESISTANT PEDIATRIC FMF PATIENTS-REAL LIFE DATA FROM THE HELIOS REGISTRY

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 179.1-180
Author(s):  
E. Sag ◽  
F. Akal ◽  
E. Atalay ◽  
U. Kaya Akca ◽  
S. Demir ◽  
...  
Keyword(s):  
Age At Onset ◽  
Real Life ◽  
First Line ◽  
Research Support ◽  
Skin Reactions ◽  
Local Skin ◽  
Extreme Stress ◽  
On Demand ◽  
Drug Registry ◽  
Treatment Onset

Background:FMF is a prototype of autoinflammatory diseases associated with excess IL1 production. Anti-IL1 treatments are the first-line alternatives in colchicine resistant/intolerant FMF patients.Objectives:We aimed to investigate the efficacy and safety of anti-IL1 treatment in pediatric FMF patients in our local (HELIOS) registry.Methods:HELIOS (Hacettepe univErsity eLectronIc research fOrmS) is a web-based biological drug registry for pediatric rheumatology patients (helios.hacettepe.edu.tr). Data were recorded at biological treatment onset (month 0), at month 6 and yearly thereafter in patients. We have analysed the clinical features, disease activity parameters, treatment responses and safety outcomes in FMF patients treated with anti-IL1 agent.Results:Forty pediatric FMF patients were included to the study group (67% female).Thirty-four patients received continous anti-IL1 treatment. The mean age at the start of the colchicine was 5.55±3.87 years. Age at onset of the anti-IL1 treatment was 11.47±5.41 with a mean follow-up duration of 3.87±1.96 years. Apart from two patients, all of them had biallelic exon-10 mutations.We have also given anti-IL1 treatment on an on-demand basis in six adolescent patients. Five of them were having very severe attacks during menstrual periods and one was having attacks during extreme stress periods along with very high CRP levels. The quality of life has markedly improved and these patients no longer reveal any CRP elevation.Anakinra was used as the first-line anti-IL1 treatment. During the last visit, six patients were treated with anakinra and 28 patients were treated with canakinumab. Anti-IL1 treatment decreased the CRP levels, number and severity of the attacks. (Figure 1.) There were three hospitalizations reported due to mild infections. Eleven patients had local skin reactions, two patients had leukopenia with anakinra and one patient had thrombocytopenia with canakinumab. We have discontinued anti-IL1 treatment until the cytopenia subsided. We have switched to on-demand therapy in one patient, started the same treatment and gradually increased the dose in the other two patients. There were no malignancy or other severe adverse reactions.Figure 1.Conclusion:Anakinra and canakinumab are efficient and safe alternatives in colchicine resistant and intolerant pediatric FMF patients. We also for the first time, report on-demand use of anti-IL1 in pediatric FMF patients. We suggest that on-demand treatment should be considered under certain circumstances where the trigger is known and short-lasting (such as menstruation and periods of extreme stress)Acknowledgments:Authors would like to thank Elif Arslanoglu Aydin, Armagan Keskin, Kubra Yuksel and Emil Aliyev for their contribution to the HELIOS registryDisclosure of Interests:Erdal Sag Grant/research support from: Novartis and SOBI financially supported the HELIOS registry during the establishment of infrastructure, Fuat Akal Grant/research support from: Novartis and SOBI financially supported the HELIOS registry during the establishment of infrastructure, Erdal Atalay Grant/research support from: Novartis and SOBI financially supported the HELIOS registry during the establishment of infrastructure, Ummusen Kaya Akca Grant/research support from: Novartis and SOBI financially supported the HELIOS registry during the establishment of infrastructure, Selcan Demir Grant/research support from: Novartis and SOBI financially supported the HELIOS registry during the establishment of infrastructure, Dilara Demirel Grant/research support from: Novartis and SOBI financially supported the HELIOS registry during the establishment of infrastructure, Ezgi Deniz Batu Grant/research support from: Novartis and SOBI financially supported the HELIOS registry during the establishment of infrastructure, Yelda Bilginer Grant/research support from: Novartis and SOBI financially supported the HELIOS registry during the establishment of infrastructure, Seza Özen Consultant of: Novartis, Pfizer, Speakers bureau: SOBI, Novartis

scholarly journals Twelve-Year Retention Rate of First-Line Tumor Necrosis Factor Inhibitors in Rheumatoid Arthritis: Real-Life Data From a Local Registry

2016 ◽  
Vol 68 (4) ◽  
pp. 432-439 ◽  
Author(s):  
Ennio Giulio Favalli ◽  
Francesca Pregnolato ◽  
Martina Biggioggero ◽  
Andrea Becciolini ◽  
Alessandra Emiliana Penatti ◽  
...  
Keyword(s):  
Rheumatoid Arthritis ◽  
Tumor Necrosis Factor ◽  
Tumor Necrosis ◽  
Retention Rate ◽  
Real Life ◽  
Tumor Necrosis Factor Inhibitors ◽  
First Line ◽  
Life Data ◽  
Real Life Data ◽  
Necrosis Factor

scholarly journals Fludarabine-Cyclophosphamide-Rituximab Treatment in Chronic Lymphocytic Leukemia, Focusing on Long Term Cytopenias Before and After the Era of Targeted Therapies

2021 ◽  
Vol 27 ◽  
Author(s):  
Róbert Szász ◽  
Béla Telek ◽  
Árpád Illés
Keyword(s):  
Targeted Therapies ◽  
Remission Rate ◽  
Real Life ◽  
Lymphocytic Leukemia ◽  
Line Treatment ◽  
First Line ◽  
Widespread Application ◽  
Real Life Data ◽  
Before And After ◽  
First Line Treatment

The widespread application of fludarabine, cyclophosphamide, and rituximab combination is limited due to its toxicity, particularly the prolonged cytopenias. The study aimed to compare the prolonged cytopenias depending on fitness and report real-life data on dose reduction measures and efficacy. According to our database, 120 and 14 patients were treated with FCR between 2011 and 2015 and between 2016 and 2019. Out of the first cohort, 34 patients were treated in subsequent lines. The complete and partial remission rate after first-line treatment was 79%, 16% in the first cohort and 86%, 14% in the second cohort, respectively; and 47%, 35% after non first-line treatment. Based on today’s standards, only 37.5% of the patients were fit for FCR. The frequency of persistent cytopenia was 14%, and it was significantly associated with fitness (χ2 (1) = 6.001, p = 0.014 for all patients). The small number of FCR treated patients after 2016 shows how the availability of targeted therapies, mostly ibrutinib, in later lines changed the first-line choice. Recently, it is recommended first-line for fit patients with mutated IGHV and no TP53 aberrations. With this narrow indication, a decrease in the frequency of persistent cytopenias is predicted.

scholarly journals Cardiovascular Risk and Statin Therapy Considerations in Women

Diagnostics ◽  
2020 ◽  
Vol 10 (7) ◽  
pp. 483 ◽  
Author(s):  
Gina Gheorghe ◽  
Peter P. Toth ◽  
Simona Bungau ◽  
Tapan Behl ◽  
Madalina Ilie ◽  
...  
Keyword(s):  
Cardiovascular Diseases ◽  
Older Women ◽  
Coronary Revascularization ◽  
Real Life ◽  
Statin Treatment ◽  
Treated Group ◽  
Fat Percentage ◽  
Teratogenic Effects ◽  
Real Life Data ◽  
Major Progress

Despite major progress in the prevention and treatment of cardiovascular diseases, women remain an underdiagnosed and insufficiently treated group, with higher hospitalization and death rates compared to men. Obesity, more frequently encountered in women, raises the risk of metabolic syndrome and cardiovascular diseases as women age. There are some differences based on sex regarding the screening, diagnosis, and treatment of dyslipidemia, as it has been observed that women are less frequently prescribed statins and, when they are, they receive lower doses, even after myocardial infarction or coronary revascularization. Real-life data show that, compared to men, women are at higher risk of non-adherence to statin treatment and are more predisposed to discontinue treatment because of side effects. Statin metabolism has some particularities in women, due to a lower glomerular filtration rate, higher body fat percentage, and overall faster statin metabolism. In women of fertile age, before initiating statin treatment, contraception methods should be discussed because statins may have teratogenic effects. Older women have a higher likelihood of polypharmacy, with greater potential for drug interactions when prescribing a statin.

Current Status of mCRC in East Europe and Middle East.

2018 ◽  
Vol 36 (4_suppl) ◽  
pp. 853-853
Author(s):  
Ozan Yazici ◽  
Gokhan Ucar ◽  
Vamvakopoulou Vasileia ◽  
Serdar Nazim Turhal ◽  
Birol Yildiz ◽  
...  
Keyword(s):  
Single Agent ◽  
Real Life ◽  
Local Relapse ◽  
Left Colon ◽  
Current Status ◽  
First Line ◽  
East Europe ◽  
Real Life Data ◽  
Wild Group

853 Background: Results obtained in trials may not necessarily translate into prolonged survival of metastatic colorectal cancer (mCRC) patients (pts) in real life.This multinational registry study aimed to evaluate the real life data effecting survival of pts with mCRC. Methods: Multinational, retrospective registry study.Those mCRC pts with at least 3 year follow up data OR died before 3 year time period:diagnosis in between 2005 – 2012. Results: Totally 208 (57%) male,156 female (43%) mCRC pts were included.Median age was 62 years (min:23 max:85).Primary locations; right (n = 71, 19.5%), transvers (n = 13, 3.6%), left colon (n = 276,75%). Sites of metastasis: liver 69.7%, lung:27.4%, local relapse %12.6, peritoneum 12%, distant lymph node 10%, CNS: 1.6%. Metastasectomy (mtc) was done in 38.5%, mtc regions: liver (22.3%), lung (4.7%), other (%11.5 ). K-N RAS and BRAF wild pts were 37.4%, 16.5% and 14.3%. Aflibercept was available for %57 of pts. MSI rate was 1.6%. Most common first line chemotherapy (FLC); Oxaliplatin (O) based combination (C) 29.9%, irinotecan (İ) C with bevacizumab (bev) 16.5%, O with bev. 16.5%. Responses to FLC; CR: 8%, PR: 30.5% stable: 28.3%, progres: 24.7%. Median OS of FLC; combination with bev: 35 months (m) (95%CI, 29.1 – 40.8), anti-EGFR:38 m (95%CI, 30.1–45.8 ),only chemotherapy: 32 m (95%CI 26.3 - 37.6), p > 0.05. Second mtc was done 10.2%.Second LC was given to %66.7 of pts. İ combination with bev (24.5%) was most common SLC. Responses to SLC; CR:4.9%, PR: 15.1% , stable:13.2% progres:29.1%. Total %31.3 and 22% of pts had third and fourth LC, most common one was single agent 5-FU. Median PFS1: 10 m (95%CI 8.3 – 11.6 ), PFS2: 7 m (95%CI 6.2 –7.7 ), PFS3: 6 m (95% CI 4.6 – 7.3 ). Median OS of all pts were 35 m (95%CI 30.7 – 39.2 ). In pts with metastasectomy compared with others median OS was; 43 m (95%CI 34.4 – 51.5) vs 29 m (95% CI 25.8 vs 32.1), respectively , p: < 0.001. Median OS in right, transvers and left colon was; 25 m (95%CI, 16.6 – 33.3), 33 m (95%CI, 8.7 – 52.7), 37 m (95%CI 32.4 – 41.5), (p = 0.25). In K-N Ras wild group median PFS; anti-EGFR:13 m (95 % CI, 9.3 - 16.6) bev:13 m (95 %CI, 4.4 - 21.5), chemo:9 m (95 % CI, 6.3 - 11.6), (p: 0.3). Conclusions: The current study demonstrated median OS and prognosis of left-transvers-right colon was consistent with the phase trials.

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