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Successful management with Janus kinase inhibitor tofacitinib in refractory juvenile dermatomyositis: a pilot study and literature review.
Yu, Z, Wang, L, Quan, M, Zhang, T, Song, H
Rheumatology (Oxford, England). 2021;(4):1700-1707
Abstract
OBJECTIVES JDM is a rare autoimmune inflammatory muscle disease with a pronounced IFN signature. Treatment for children with JDM has improved over the years with the use of steroids and immunosuppressive agents. However, there remains a subset of children who have refractory disease. Janus kinase and type I IFN signalling production are suspected to contribute to the pathogenesis of JDM. Our pilot study investigated the use of tofacitinib, a Janus kinase inhibitor, in refractory JDM cases to provide new therapeutic options for better treatment. METHODS Refractory JDM was defined as patients who failed two or more steroid sparing agents or high-dose steroids. Tofacitinib was given to three refractory JDM patients with a dose of 5 mg twice per day for at least 6 months. Core set measures defined by Pediatric Rheumatology International Trials Organization were evaluated at month 0, 3 and 6 along with other systemic evaluations. A literature review was conducted to identify all the cases using Janus kinase inhibitors in JDM. RESULTS All three subjects tolerated and responded well to tofacitinib with significant improvement in Child Myositis Assessment Scale, manual muscle testing-8, physician global disease activity and inflammatory indices without occurrence of severe adverse events. CONCLUSION This pilot study showed improvement of muscle strength, resolution of cutaneous lesions, increased daily quality of life and successful tapering of steroids when tofacitinib used in selected cases. Tofacitinib can be considered with caution when treating refractory JDM cases. Further randomized controlled trials are warranted to assess its efficacy in JDM.
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2.
Occurrence of abscesses during treatment with pazopanib in metastatic renal cancer: a case report.
Puliafito, I, Russo, A, Sciacca, D, Puglisi, C, Giuffrida, D
Journal of medical case reports. 2020;(1):7
Abstract
BACKGROUND Pazopanib is a multitarget tyrosine kinase inhibitor used in the treatment of renal cancer and soft tissue sarcoma. Its use is commonly associated with a number of side effects, such as hemorrhagic diathesis, neutropenia, leukopenia, thrombocytopenia, nausea, vomiting, abdominal pain, increased serum aspartate aminotransferase, increased serum alanine aminotransferase, decreased serum glucose, increased serum bilirubin, decreased serum phosphate and magnesium, fatigue, hypertension, diarrhea, anorexia, proteinuria, and hypothyroidism. Abscesses of metastases caused by pazopanib administration are rarely reported in the literature. CASE PRESENTATION We report a case of abscesses of lung metastases related to pazopanib in a patient with metastatic renal cancer. The patient was a 53-year-old Caucasian man who developed abscesses of lung metastases during the first 3 months of treatment with pazopanib. The abscesses resolved after 1 month by stopping pazopanib and administering adequate antibiotic therapy. CONCLUSIONS We conclude that abscesses of metastases could be a rare side effect occurring during treatment with pazopanib in patients with renal cancer.
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3.
Recurrent multiple CNS hemangioblastomas with VHL disease treated with pazopanib: a case report and literature review.
Migliorini, D, Haller, S, Merkler, D, Pugliesi-Rinaldi, A, Koka, A, Schaller, K, Leemann, B, Dietrich, PY
CNS oncology. 2015;(6):387-92
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Abstract
Hemangioblastoma is a rare benign neoplasm, accounting for less than 2% of all primitive brain tumors. It may arise sporadically in a solitary form, or associated with Von Hippel-Lindau (VHL) disease with multiple tumors. Surgery is the mainstay treatment, but management is challenging in case of recurrent and/or multiple tumors. VHL protein is defective in both forms of hemangioblastoma, leading to the accumulation of hypoxia-inducible factor, stimulating angiogenesis via VEGF and PDGF mainly. Here, we report a 37-year-old woman's case with recurrent and rapidly progressive VHL-associated hemangioblastomas, causing severe disability. She was treated 24 months with pazopanib, a multityrosine kinase inhibitor (TKI) targeting VEGF and PDGF-β pathways. Despite moderate radiological changes, progressive improvement in her clinical condition persisting over 3 years was observed. Inhibiting angiogenesis is a therapeutic option that may improve the quality of life and the autonomy of VHL patients disabled with multiple hemangioblastomas.
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4.
Low-dose imatinib in the treatment of severe systemic sclerosis: a case series of six Chinese patients and literature review.
Guo, L, Chen, XX, Gu, YY, Zou, HJ, Ye, S
Clinical rheumatology. 2012;(9):1395-400
Abstract
Systemic sclerosis (SSc) is a progressive fibrotic disorder with no legitimate effective treatment. Several clinical trials had investigated imatinib mesylate with a target dose of 400∼600 mg/day on SSc, and the efficacy is controversial with a generally poor tolerability. We herein reported six female Chinese patients with SSc administered with low-dose imatinib (200 mg/day) for a median of 23 months (10∼30 months). Patients displayed a decreased modified Rodman skin scores (mRSS) by a mean of 6.29 points after 6 months of treatment. Three patients who completed 2 years of treatment achieved a reduction of mRSS by 8, 18, and 30.5 points, respectively. Pulmonary function was improved or stabilized in two patients with interstitial lung disease. Severe gastrointestinal involvement in one patient was attenuated in terms of discontinuation of total parenteral nutrition and restoration of the serum albumin level. Imatinib was well-tolerated in general, although there were two severe adverse events: a bone fracture and a cerebral hemorrhage in two individuals. Both the adverse events were probably not directly related to imatinib and were recovered uneventfully. Our limited data, along with the review of the literature, suggested that low-dose imatinib might be effective and better tolerated in severe SSc that deserves further study.
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Medication-induced periostitis in lung transplant patients: periostitis deformans revisited.
Chen, L, Mulligan, ME
Skeletal radiology. 2011;(2):143-8
Abstract
We report five cases of diffuse periostitis resembling hypertrophic osteoarthropathy and perostitis deformans in lung transplantation patients on chronic voriconazole, a fluoride-containing compound. Although drug-related periostitis has long been known, the association of lung transplant medication with periostitis was only recently introduced in the literature. To our knowledge, imaging findings have not been fully characterized in the radiology literature. Imaging features along with clinical history help to distinguish this benign condition from other disease entities. In this article, we review the current literature and illustrate the variety of imaging characteristics of this entity so that interpreting radiologists can make accurate diagnoses and avoid unnecessary work up.
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Case report. Hyperlipoproteinaemia(a): which is the optimal therapy? A case report.
Lupattelli, G, Roscini, AR, Siepi, D, Mannarino, E
Journal of clinical pharmacy and therapeutics. 2010;(5):613-5
Abstract
This case report presents the clinical history of a patient with elevated lipoprotein(a) and small size isoform, associated with mixed hyperlipaemia, which was probably familial combined hyperlipaemia. After premature myocardial infarction, the subject was treated with fibrates. Niacin was started after recurrence. One year ago, after another episode of acute coronary syndrome, rosuvastatin was added to niacin. The atherogenicity of this lipid disorder, along with the different options for therapy is discussed.
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Rosuvastatin-induced rhabdomyolysis probably via CYP2C9 saturation.
Gallelli, L, Ferraro, M, Spagnuolo, V, Rende, P, Mauro, GF, De Sarro, G
Drug metabolism and drug interactions. 2009;(1):83-7
Abstract
A 66 year-old woman with no history of renal or liver disease presented with progressive asthenia and diffuse myalgia. She cited 5 months history of mild hyperlipidemia under treatment with rosuvastatin (10 mg/day). Clinical examination documented both an increase in liver size and proximal muscle weakness, with difficulty in raising arms above the head. Blood tests showed the presence of renal, liver and muscle failure, with no evidence of virological, immunological or haematological diseases. Rosuvastatin treatment was stopped and blood values normalised within five days; but because of an increase in cholesterol plasma levels, rosuvastatin (10 mg/day) was restarted. Two days later, the patient returned to our observation due to the development of asthenia and muscle weakness, with an increase in creatine phosphokinase, 12,165 U/l. Rosuvastatin was discontinued and replaced with pravastatin (40 mg/day) with a complete resolution of clinical and laboratory findings in about six days. Our patient was taking rosuvastatin, warfarin and telmisartan, which are metabolised by CYP2C9; we therefore hypothesised that the rosuvastatin-induced rhabdomyolysis was probably by CYP2C9 enzyme saturation.
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[Gastrointestinal stromal tumor of small intestine associated with lymph node metastasis: a report of 2 cases with review of literatures].
Kong, M, Wang, YL, Xu, LJ, Teng, XD
Zhonghua bing li xue za zhi = Chinese journal of pathology. 2009;(9):617-20
Abstract
OBJECTIVE To study the clinicopathologic features of gastrointestinal stromal tumor (GIST) of small intestine with lymph node metastasis and evaluate the respond to imatinib mesylate (Glivec) therapy. METHODS Two cases of GIST of small intestine associated with lymph node metastasis were collected and investigated by light microscopy and immunohistochemistry. Mutation in exon 9, 11 and of c-kit gene were analyzed by polymerase chain reaction and DNA sequencing. RESULTS The cases presented as small intestinal mass of irregular shape. Histologically, the tumors consisted of epithelioid and spindled cells, with areas of coagulative necrosis and hemorrhage. The mitotic count measured about 2 per 50 high-power fields. Immunohistochemical study showed that the tumor cells were diffusely distributed and strongly positive for CD117. Mutation analysis revealed that case 1 had an in-frame deletion of 11 amino-acid residues corresponding to 559-569 and carried two missense mutations involving codons 570, 571 in exon 11 of c-kit gene. Case 2 revealed an in-frame deletion involved condons 559-565 in exon 11 of c-kit gene. These two cases were all underwent primary chemotherapy with imatinib mesylate and without new tumor was found during follow-up periods (18, 26 months) after operation. CONCLUSIONS GIST with nodal metastasis is very rare and needs to be distinguished from other soft tissue sarcomas occurring in this site. The responsiveness to imatinib mesylate therapy correlates with the mutation status of c-kit gene.
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Imatinib-induced tumor lysis syndrome: report of a case and review of the literature.
Chang, H, Shih, LY
Chang Gung medical journal. 2008;(5):510-4
Abstract
Imatinib is a selective tyrosine kinase inhibitor which acts on breakpoint cluster region-Abelson fusion gene (BCR-ABL) positive leukemia including all phases of chronic myeloid leukemia and acute lymphoblastic leukemia. It may induce rapid apoptosis and subsequent tumor lysis syndrome. Only 3 cases of imatinib-induced tumor lysis syndrome have been reported. We herein described an additional patient with BCR-ABL (ela2) positive acute lymphoblastic leukemia who developed tumor lysis syndrome after 10-day treatment with imatinib. Experience in the current case suggests that preventive measures for tumor lysis syndrome, including allopurinol and hydration, should be taken for patients with high leukemia burden who receive imatinib therapy, and parameters of tumor lysis should be monitored in the early phase of therapy.
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10.
A multimodal, evidence-based approach to achieve lipid targets in the treatment of antiretroviral-associated dyslipidemia: case report and review of the literature.
Bain, AM, White, EA, Rutherford, WS, Rahman, AP, Busti, AJ
Pharmacotherapy. 2008;(7):932-8
Abstract
Metabolic abnormalities associated with the treatment of human immunodeficiency virus (HIV) infection are well-recognized problems that increase cardiovascular risk. As a result of the complexity of treating both HIV- and antiretroviral-related comorbidities, strategies that improve adverse drug events while maintaining viral control are in critical need. Although guidelines have somewhat helped in the general approach and in first-line strategies for managing dyslipidemia in patients receiving antiretrovirals, a paucity of data exist to guide clinicians in treating patients whose conditions are refractory to first-line options or who are at substantial risk for cardiovascular events. Further complicating the choice of lipid-lowering strategy is the lack of randomized controlled data from the HIV-affected population and a concern about clinically significant drug-drug interactions. We describe an HIV-infected patient with efavirenz-associated dyslipidemia at very high cardiovascular risk who had not achieved his primary or secondary lipid goals despite 2 years of treatment in a lipid specialty clinic. Lipid control was accomplished in 10 weeks with a targeted, stepwise approach of switching efavirenz to nevirapine, followed by rosuvastatin 20 mg/day, which was sustained for at least 10 months. Of most importance, this outcome was achieved without any clinically significant alteration in virologic or immunologic control. This case report highlights the potential for a pharmacist-guided, multistep approach that addresses HIV-related dyslipidemia and incorporates the pharmacokinetic literature to guide lipid-lowering therapy and promote the attainment of goals based on current standards of care.