A prospective open-label study of mycophenolate mofetil for the treatment of diffuse systemic sclerosis

Derk, Chris T.; Grace, Elizabeth; Shenin, Max; Naik, Manisha; Schulz, Steffan W.; Xiong, Jian

doi:10.1093/rheumatology/kep295

Cited by 93 publications

(64 citation statements)

References 16 publications

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“…The observed degree of improvement of mRSS in the study described here correlates well with prior studies by Derk et al (19) and Le et al (20) who found a reduction of 14.1 and 7.6 points in the mRSS of patients treated with MMF, respectively. Given the heterogeneous SSc course, the greater than 10 point mRSS improvement and the 23% reduction in affected BSA observed here are likely clinically significant for this population of previously untreated patients with SSc of 11 recent onset with diffuse cutaneous involvement and usually progressive clinical course.…”

Section: Discussionsupporting

confidence: 91%

“…Here, we describe a prospective observational study of MMF for the treatment of SSc in patients with recent onset of progressive diffuse cutaneous involvement. In contrast with other published studies (16)(17)(18)(19)(20) the patients included in our study had not received any previous immunosuppresive, immunomodulating, or antifibrotic therapy, and none of the patients received any other disease modifying therapy during the study.…”

Section: Introductionmentioning

confidence: 89%

“…4 Several studies including a recent assessment of 98 patients have described encouraging results of MMF treatment for SSc (16)(17)(18)(19)(20). In contrast, a recent observational trial failed to show significant differences between four different therapeutic approaches, three of which included MMF (21).…”

Section: Introductionmentioning

confidence: 99%

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A Prospective Observational Study of Mycophenolate Mofetil Treatment in Progressive Diffuse Cutaneous Systemic Sclerosis of Recent Onset

Mendoza¹,

Nagle²,

Lee³

et al. 2012

J Rheumatol

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Objective.A prospective observational study of mycophenolate mofetil (MMF) treatment in patients with diffuse progressive cutaneous systemic sclerosis (SSc) of recent onset.Methods.Twenty-five previously untreated consecutive patients with recent-onset (< 24 mo) diffuse progressive cutaneous SSc received MMF as the only disease-modifying therapy. Modified Rodnan skin score (mRSS) and affected body surface area (BSA) were compared from initiation of MMF to study end. Pulmonary function tests performed at the same institution before therapy and at study end were available in 15 patients. Histopathology and real-time PCR assessment of fibrosis-related gene expression were performed before and after treatment in skin biopsies from 3 patients.Results.At 18.2 ± 8.73 months of MMF therapy (median 2000 mg/day) the mRSS decreased from 24.56 ± 8.62 to 14.52 ± 10.9 (p = 0.0004) and the affected BSA from 36% ± 16% to 14% ± 13.3% (p = 0.00001). Pulmonary function tests remained stable from initiation of MMF to the end of the study. Skin histopathology showed a remarkable reduction in accumulation of fibrotic tissue. Real-time PCR of skin biopsies demonstrated a marked decrease in expression of fibrosis-related genes.Conclusion.Patients with diffuse progressive cutaneous SSc of recent onset treated with MMF experienced marked improvement in skin involvement and stabilization of pulmonary function. Skin biopsies from 3 patients demonstrated histopathological improvement and decreased expression of fibrosis-related genes.

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Section: Discussionsupporting

confidence: 91%

Section: Introductionmentioning

confidence: 89%

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A Prospective Observational Study of Mycophenolate Mofetil Treatment in Progressive Diffuse Cutaneous Systemic Sclerosis of Recent Onset

Mendoza¹,

Nagle²,

Lee³

et al. 2012

J Rheumatol

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“…Aunque en términos generales la evidencia está a favor de usarlo, en muchas ocasiones este medicamento se administró combinado con otros inmunosupresoras lo que impide establecer el beneficio aislado del micofenolato (34)(35)(36)(37)(38)(39)(40)(41)(42).…”

Section: ¿Cómo Tratar?unclassified

Enfermedad pulmonar intersticial en pacientes con esclerosis sistémica. Revisión narrativa de la literatura

Álvarez-Barreneche

Velásquez-Franco

Navas

2017

iatreia

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RESUMENIntroducción: la esclerosis sistémica es una enfermedad autoinmune crónica, caracterizada por la tríada de vasculopatía de pequeños vasos, activación del sistema inmune y aumento de los depósitos de matriz extracelular tanto en la piel como en los órganos internos. En los últimos años, el compromiso pulmonar ha cobrado gran importancia, pasando a ser la primera causa de muerte, dada la disminución de la mortalidad por crisis renales con el advenimiento de los inhibidores de la enzima convertidora de angiotensina. La afección pulmonar puede ocurrir como hipertensión o enfermedad intersticial difusa; esta última es más común en pacientes con la variedad generalizada, en los primeros tres años de la enfermedad. Tiene mal pronóstico si no se inicia el tratamiento, cuya meta es detener el deterioro de la función pulmonar. Entre los tratamientos disponibles, la ciclofosfamida cuenta con la mejor evidencia, pero se están estudiando, con resultados preliminares satisfactorios, otros tratamientos como micofenolato mofetil, rituximab, trasplante autólogo de células hematopoyéticas y trasplante de pulmón.Objetivo: describir, de acuerdo con lo reportado en la literatura, la epidemiología, la fisiopatología, los métodos diagnósticos y el tratamiento de la enfermedad pulmonar intersticial en pacientes con esclerosis sistémica.Métodos: revisión estructurada, no sistemática de la literatura, enfocada en los aspectos de interés, mencionados y que incluyó 52 artículos.

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“…Although the toxicity of MMF appears tolerable, the longterm toxicity of MMF in this setting remains unknown. Whether MMF has additional disease-modifying benefits such as in improving the cutaneous involvement of SSc remains unclear 13,14,15,16 . Finally, as the authors note, it is unclear how long patients should be treated with MMF to avoid progression of lung fibrosis.…”

mentioning

confidence: 99%

Can Large Simple Trials Help Us Understand When and How to Use Generic Drugs for Uncommon Diseases?

Molitor

DeMets²

2013

J Rheumatol

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A prospective open-label study of mycophenolate mofetil for the treatment of diffuse systemic sclerosis

Cited by 93 publications

References 16 publications

A Prospective Observational Study of Mycophenolate Mofetil Treatment in Progressive Diffuse Cutaneous Systemic Sclerosis of Recent Onset

A Prospective Observational Study of Mycophenolate Mofetil Treatment in Progressive Diffuse Cutaneous Systemic Sclerosis of Recent Onset

Enfermedad pulmonar intersticial en pacientes con esclerosis sistémica. Revisión narrativa de la literatura

Can Large Simple Trials Help Us Understand When and How to Use Generic Drugs for Uncommon Diseases?

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