scholarly journals The Efficacy of Baricitinib in Real-World Patients with Refractory or Severe Juvenile Dermatomyositis:A Monocentric Retrospective Study

Author(s):  
Zhaoling Wang ◽  
Qi Zheng ◽  
XiSheng Xu ◽  
Meiping Lu

Abstract Objective To evaluate the efficacy and safety of low dose baricitinib in children with refractory or severe juvenile dermatomyositis (JDM) in a real-world setting. Methods A monocentric retrospective real-world study was conducted, in which fourteen refractory and one severe newly diagnosed JDM patients were included. These patients were all treated by low dose baricitinib (below the recommended dose) combined with corticosteroids and or immunosuppressive agents. Clinical data were collected at the baseline and 4, 12, 24 weeks after baricitinib implication. Treatment response (complete response (CR), Partial response (PR) and non-response (NR)) was evaluated using both the Paediatric Rheumatology International Trials Organization (PRINTO) remission criteria and skin Disease Activity Score (DAS). All the adverse events (AEs) were recorded. Results After baricitinib treatment, all 15 patients showed improvement of skin involvement, including 14 patients with recurrent skin rashes and one newly diagnosed JDM. Calcinosis stabilized in two patients (2/3) and partially regressed in one. Four patients (4/15) had interstitial lung disease (ILD), which normalized in one, improved in two and stabilized in one. One patient complicated with macrophage activation syndrome (MAS) achieved clinical remission. CR was achieved in 3/15 patients, ranging from 4 to 12 weeks after baricitinib initiation. Five patients (5/15) got PR 4 to 24 weeks after baricitinib use. Daily steroid dosage was decreased from 0.632 mg/kg to 0.357 mg/kg (P = 0.043) at 24 weeks in all responders. However, there was no statistically difference in muscle improvement. One patient was stopped using baricitinib because of varicella zoster virus infection, while no other serious side effect was observed in this study. Conclusion Low dose baricitinib had efficacy and was safe to applied in refractory or severe JDM patients, especially for recurrent skin rashes. Baricitinib may also be helpful for JDM complicated with ILD and MAS.

2021 ◽  
Author(s):  
Zhaoling Wang ◽  
Qi Zheng ◽  
XiSheng Xu ◽  
MeiPing Lu

Abstract Objective To evaluate the efficacy and safety of low dose baricitinib in children with refractory or severe juvenile dermatomyositis(JDM) in a real-world setting. Methods A monocentric retrospective real-world study was conducted, in which fourteen refractory and one severe newly diagnosed JDM patients were included. These patients were all treated by low dose baricitinib (below the recommended dose) combined with corticosteroids and or immunosuppressive agents. Clinical data were collected at the baseline and 4, 12, 24 weeks after baricitinib implication. Treatment response (complete response, CR, Partial response, PR and non-response,NR) was evaluated using both the Paediatric Rheumatology International Trials Organization (PRINTO) remission criteria and skin Disease Activity Score (DAS). All the adverse events (AEs) were recorded. Results After baricitinib treatment, all 15 patients showed improvement of skin involvement, including 14 patients with recurrent skin rashes and one newly diagnosed JDM. Calcinosis stabilized in two patients (2/3) and partially regressed in one. Four patients (4/15) had interstitial lung disease (ILD), which normalized in one, improved in two and stabilized in one. One patient complicated with macrophage activation syndrome (MAS) achieved clinical remission. CR was achieved in 3/15 patients, ranging from 4 to 12 weeks after baricitinib initiation. Five patients (5/15) got PR 4 to 24 weeks after baricitinib use. Daily steroid dosage was decreased from 0.632 mg/kg to 0.357 mg/kg (P = 0.043) at 24 weeks in all responders. However, there was no statistically difference in muscle improvement. One patient was stopped using baricitinib because of varicella zoster virus infection, while no other serious side effect was observed in this study. Conclusion Low dose baricitinib had efficacy and was safe to applied in refractory or severe JDM patients, especially for recurrent skin rashes. Baricitinib may also be helpful for JDM complicated with ILD and MAS.


2017 ◽  
Vol 35 (8_suppl) ◽  
pp. 247-247
Author(s):  
Bruce Jeffrey Dezube ◽  
Jill Bell ◽  
Aaron Galaznik ◽  
Eileen Farrelly ◽  
Marlo Blazer ◽  
...  

247 Background: Treatment decisions in MDS are based on a prognostic scoring system that divides pts into five distinct risk categories (NCCN 2016). Treatment guidelines for HR MDS pts include hypomethylating agents (HMAs) alone (azacitidine & decitabine), high-intensity induction chemotherapy (IC), & stem cell transplant (SCT) alone or after HMAs. Limited information is available on how these recommendations are applied in practice. This study evaluated the real-world treatment of HR MDS pts. Methods: Newly diagnosed HR MDS pts who were ≥18 years old & initiated first-line therapy (1LT) were retrospectively identified from a large United States EMR database between 1/1/2008 & 7/31/2015. As complete cytogenetics were not available in the database, HR was based on: ICD coding: ≥1 inpatient claim with an HR MDS ICD-9/10 code (ICD-9 code: 238.73; ICD-10 codes: D46.20, D46.21, D46.22), or ≥2 outpatient claims with an MDS ICD-9/10 code, or an adapted HR MDS algorithm (NCCN Guidelines in Oncology for MDS v.1.2016; Greenberg, et al. Blood. 2012;120:2454-65; Schanz et al. J Clin Oncol. 2012;30:820-9). The first MDS claim served as the index date. 1LT was defined as an MDS-specific treatment initiated on or after the index date. Pts were followed until death, progression to acute myeloid leukemia (AML), loss to follow-up, or end of study (9/30/2015). Results: 720 newly diagnosed HR MDS pts were identified; of these, 229 (32%) pts received MDS-specific treatment. Median time to treatment was 22 days (interquartile range [IQR]: 10, 74). HMAs were the most common agents in the 1LT with 60% (n= 138) & 24% (n=54) receiving azacitidine & decitabine, respectively. Lenalidomide was used in 7.4% of pts (n=17), 4.8% received SCT alone (n=11), & 3.9% (n=9) received IC. At median follow-up of 9.4 months (IQR: 4.3, 18.4), 38% (n=86) died & 28% (n=63) progressed to AML. Conclusions: Despite guidelines, most HR MDS pts in a real-world setting were not treated with MDS-specific treatment. Among treated pts, 1LT with HMAs & azacitidine predominated. Subsequent research is needed to understand reasons for lack of treatment.


2021 ◽  
Vol 9 (1) ◽  
pp. 34-37
Author(s):  
Fauzia Sobhan ◽  
Khaza Moiz ◽  
Naima Siddiquee ◽  
Aditi Debnath ◽  
Fazlul Haque ◽  
...  

Rheumatoid arthritis is a painful debilitating joint disease with the proliferation of the synovium and progressive erosion of cartilage and bone. Methotrexate (MTX) has been used for the treatment of Rheumatoid Arthritis (RA) for about 3 decades. It is most effective and commonly used Disease Modifying Antirheumatic Drugs (DMARDS) because it improves symptoms, signs, disease activity and functions. This study was done from January 2016 to December 2016 for a period of 1 year under Physical Medicine and Rehabilitation department, Jalalabad Ragib-Rabeya Medical College (JRRMC), Sylhet. Here we treated the patients with low dose methotrexate. Study was done to see the effect of low dose MTX in newly diagnosed RA patients by clinical examination and DAS 28. DAS stands for disease activity score and 28 joints that are examined in this assessment. The result concluded low dose MTX improves the symptoms, signs, disease activity and functions of the patients. Bangladesh Crit Care J March 2021; 9(1): 34-37


2020 ◽  
Vol 16 (14) ◽  
pp. 939-953 ◽  
Author(s):  
Meinolf Karthaus ◽  
Gülten Oskay-Özcelik ◽  
Pia Wülfing ◽  
Carsten Hielscher ◽  
Dagmar Guth ◽  
...  

Aim: To determine quality of life, effectiveness and safety of oral netupitant-palonosetron (NEPA)–based antiemetic prophylaxis in the real-world setting. Materials & methods: Prospective, noninterventional study in adults receiving highly or moderately emetogenic chemotherapy and NEPA for three cycles. NEPA was administered per summary of product characteristics. Results: A total of 2429 patients enrolled, 2173 were evaluable. ‘No impact on daily life’ due to vomiting was reported by 85%/82% of patients in the highly emetogenic chemotherapy/moderately emetogenic chemotherapy groups in cycle 1, with rates of 54%/59% for nausea. Overall, complete response rate was 89%/87%/75% in the acute/delayed/overall phases. NEPA was well tolerated. Conclusion: NEPA had beneficial effects on the quality of life of a heterogeneous group of cancer patients and was safe and effective in the real-world setting.


Author(s):  
Vivek Kumar Verma ◽  
Vidya Sagar Ram ◽  
Prem Shanker Singh ◽  
Manoj Kumar ◽  
Sankalp Awasthi ◽  
...  

Background: Herpes zoster (HZ) occurs due to reactivation of latent Varicella zoster virus infection and affects in dermatomal pattern. HZ affects elderly and immunocompromised population. Some earlier studies shows that HZ is common in diabetes patients. Our aim was to find out incidence of diabetes mellitus in patients with HZ.Methods: Study was done on newly diagnosed HZ patients attending out door of UPUMS Saifai. Inclusion criteria include newly diagnosed case of HZ without previous history of diabetes. Patients with known immunocompromised state like HIV infection, corticosteroid therapy, chemotherapy, neoplastic disease etc were excluded. Fasting, post-prandial blood sugar and HbA1C of all patients done.Results: 22.54% patients with HZ had diabetes and 7.75% patients had impaired glucose tolerance at presentation. Undiagnosed diabetes is common in HZ patients.Conclusions: Our study indicates that incidence of undiagnosed diabetes is high among HZ patients and hence routine screening for diabetes should be done in all HZ patients.


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