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1.
Pediatr Rheumatol Online J ; 21(1): 2, 2023 Jan 04.
Artigo em Inglês | MEDLINE | ID: mdl-36600239

RESUMO

OBJECTIVES: Familial Mediterranean Fever (FMF) patients are required to adhere to a life-long treatment with colchicine, primarily for preventing amyloidosis. As some patients may be asymptomatic for long periods of time, it remains unclear whether it is possible to discontinue colchicine treatment in a selective group of patients. We aimed to identify predictive characteristics for a successful cessation of colchicine therapy. METHODS: Out of 646 FMF pediatric patients followed in our referral FMF clinic, colchicine treatment was discontinued in 51 patients. In this study we compared the genetic, demographic, and clinical characteristics between patients for whom a successful cessation of therapy was made (Group 1; n = 21) and patients for whom cessation of therapy was deemed a failure (Group 2; n = 30) and consequently had to resume colchicine therapy. RESULTS: Patients for whom a successful cessation of therapy was achieved had no biallelic pathogenic MEFV mutations, were less likely to have "severe attacks" (two or more FMF characteristic symptoms) (24% vs 80%; P = 0.000067) and did not require higher than 1 mg/day of colchicine, prior to the drug cessation. Remission duration under colchicine treatment was significantly higher in group 1 compared with group 2 (4.36 years ±2.12 vs 2.53 years ±2; P = 0.0036). CONCLUSION: This study supports the concept of colchicine free remission in a minority of FMF patients (3%). Holding treatment, under close monitoring, may be reasonable when selecting the appropriate patients.


Assuntos
Amiloidose , Colchicina , Febre Familiar do Mediterrâneo , Criança , Humanos , Amiloidose/tratamento farmacológico , Amiloidose/genética , Colchicina/uso terapêutico , Febre Familiar do Mediterrâneo/tratamento farmacológico , Febre Familiar do Mediterrâneo/genética , Febre Familiar do Mediterrâneo/diagnóstico , Pirina/genética
3.
Leuk Lymphoma ; 61(11): 2645-2651, 2020 11.
Artigo em Inglês | MEDLINE | ID: mdl-32643497

RESUMO

Progression of disease within 24 months of initial therapy (POD24) has previously been identified as a predictor of reduced overall survival (OS) for patients with follicular lymphoma (FL). Here we attempt to validate this finding in a retrospective cohort and understand whether the method by which progression is determined, clinically or radiographically, influences POD24 robustness. We reviewed records of 635 patients with FL and included 317 patients in our analysis. POD24 occurred in 21.5% of patients and it was evident that OS was significantly lower in the POD24 group. In multivariate analysis both POD24 and FLIPI were independently associated with inferior OS. POD24 that was detected by incidental routine imaging did not predict reduced OS as opposed to progression that was detected clinically. Although surveillance imaging is generally discouraged in FL, it still is a routine practice by many physicians, and therefore our findings are of significant clinical implications.


Assuntos
Linfoma Folicular , Protocolos de Quimioterapia Combinada Antineoplásica , Estudos de Coortes , Progressão da Doença , Humanos , Linfoma Folicular/tratamento farmacológico , Prognóstico , Estudos Retrospectivos
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