Evaluation of the Effectiveness and Tolerability of Mycophenolate Mofetil and Mycophenolic Acid for the Treatment of Morphea

Arthur, Megan; Fett, Nicole; Latour, Emile; Jacobe, Heidi; Kunzler, Elaine; Florez-Pollack, Stephanie; Houser, Jacob; Sharma, Shivani; Prasad, Smriti; Femia, Alisa; Stern, Marleigh J.; Pappas‐Taffer, Lisa; Gaffney, Rebecca G.; Fernandez, Anthony P.; Knabel, Daniel; Cardones, Adela R.; Leung, Nicole; Laumann, Anne E.; Yu, Jack; Zhao, Jeffrey; Vleugels, Ruth Ann; Tkachenko, Elizabeth; Lo, Kelly

doi:10.1001/jamadermatol.2020.0035

Cited by 28 publications

(29 citation statements)

References 25 publications

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“…There is a potential role for hydroxychloroquine in the treatment of morphea, with a retrospective study showing a majority of patients having at least a greater than 50% response to 6 months of therapy 7 . A retrospective study of mycophenolate in the treatment of morphea showed a majority of patients to have stable or improved disease within 3–6 months of therapy 8 . However, further studies are needed to better elucidate the efficacy of these therapies in radiation‐induced morphea.…”

Section: Discussionmentioning

confidence: 99%

“…7 A retrospective study of mycophenolate in the treatment of morphea showed a majority of patients to have stable or improved disease within 3-6 months of therapy. 8 However, further studies are needed to better elucidate the efficacy of these therapies in radiation-induced morphea. Familiarity with this condition as sequelae of radiation therapy can lead to earlier recognition and dermatologic referral, improving the outcomes and quality of life of these patients.…”

Section: Discussionmentioning

confidence: 99%

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Radiation‐induced morphea following irradiation of osteoblastic metastases

Sugerik

Farooq²,

Fraga‐Braghiroli³

2022

JEADV Clinical Practice

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This case describes a patient with development of radiation-induced morphea after irradiation to the femurs for osteoblastic metastases of breast cancer. This rare and underdiagnosed complication of radiation therapy is an important contributor to morbidity in oncologic patients. Increased awareness of this condition will allow earlier dermatologic consultation and may improve cosmetic and functional outcomes.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Radiation‐induced morphea following irradiation of osteoblastic metastases

Sugerik

Farooq²,

Fraga‐Braghiroli³

2022

JEADV Clinical Practice

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“…A case series of MMF in 22 patients with JLS compared to 47 on MTX showed no significant difference in relapse-free survival between the groups although MMF appeared to induce more persistent remission, and MMF was well tolerated 40 . In a study of 77 participants (all 16 years or older), MMF was well tolerated with 35% achieving disease remission 52 . Both studies concluded that further controlled studies are needed.…”

Section: Discussionmentioning

confidence: 99%

Prior elicitation of the efficacy and tolerability of Methotrexate and Mycophenolate Mofetil in Juvenile Localised Scleroderma

et al. 2021

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Background Evidence is lacking for safe and effective treatments for juvenile localised scleroderma (JLS). Methotrexate (MTX) is commonly used first line and mycophenolate mofetil (MMF) second line, despite a limited evidence base. A head to head trial of these two medications would provide data on relative efficacy and tolerability. However, a frequentist approach is difficult to deliver in JLS, because of the numbers needed to sufficiently power a trial. A Bayesian approach could be considered. Methods An international consensus meeting was convened including an elicitation exercise where opinion was sought on the relative efficacy and tolerability of MTX compared to MMF to produce prior distributions for a future Bayesian trial. Secondary aims were to achieve consensus agreement on critical aspects of a future trial. Results An international group of 12 clinical experts participated. Opinion suggested superior efficacy and tolerability of MMF compared to MTX; where most likely value of efficacy of MMF was 0.70 (95% confidence interval (CI) 0.34-0.90) and of MTX was 0.68 (95% CI 0.41-0.8). The most likely value of tolerability of MMF was 0.77 (95% CI 0.3-0.94) and of MTX was 0.62 (95% CI 0.32-0.84). The wider CI for MMF highlights that experts were less sure about relative efficacy and tolerability of MMF compared to MTX. Despite using a Bayesian approach, power calculations still produced a total sample size of 240 participants, reflecting the uncertainty amongst experts about the performance of MMF. Conclusions Key factors have been defined regarding the design of a future Bayesian approach clinical trial including elicitation of prior opinion of the efficacy and tolerability of MTX and MMF in JLS. Combining further efficacy data on MTX and MMF with prior opinion could potentially reduce the pre-trial uncertainty so that, when combined with smaller trial sample sizes a compelling evidence base is available.

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“…This is supported by three retrospective cohort studies with a total of 94 patients who received second‐line MMF therapy, usually concomitant with additional treatment 24–26 . Of the patients with a treatment duration of at least 9 months, 25% had stable and 61% had improved disease 24 . However, cases refractory to MMF have been observed, and the treatment of recalcitrant morphoea remains largely empirical.…”

Section: Second‐line Therapy For Difficult‐to‐treat Casesmentioning

confidence: 91%

Diagnosis and management of morphoea in children: an overview

Weibel

2021

Clin Exp Dermatol

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Summary Paediatric morphoea (localized scleroderma) is an inflammatory sclerosing disorder of the skin and subcutis associated with tissue atrophy. It is thought that the disease develops on the background of genetic predisposition (e.g. mosaicism for the common linear variant) initiated by various trigger factors, and that detected autoantibodies and inflammatory cytokines represent secondary epiphenomena. In contrast to the common belief that morphoea is a benign self‐limiting disorder, long‐term data indicate that its chronicity, relapsing nature and extracutaneous complications lead to significant morbidity, particularly when the disease starts in early childhood. Early recognition may be challenging, and the most important clinical clues are band‐like distribution, atrophy of underlying tissue, skin sclerosis, and localized loss of body/scalp hair, eyelashes or eyebrows. Extracutaneous manifestations occur in up to 20% of patients, with arthritis/arthralgia and neurological symptoms being most frequently observed, followed by ophthalmological complications such as uveitis. Corticosteroids and methotrexate are highly effective as first‐line therapy in morphoea, leading to partial reversal of skin manifestations. However, the development of atrophy is not sufficiently prevented by standard therapy. There is a relapse rate of 25%–48% within the first years after stopping treatment, thus long‐term follow‐up is warranted. Mycophenolate mofetil seems to be a beneficial second‐line therapy, and a new drug, abatacept, also seems to be a promising and well‐tolerated second‐line treatment option. Additionally, autologous fat injections are beneficial and may be used as an adjunct to ongoing therapy.

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Evaluation of the Effectiveness and Tolerability of Mycophenolate Mofetil and Mycophenolic Acid for the Treatment of Morphea

Cited by 28 publications

References 25 publications

Radiation‐induced morphea following irradiation of osteoblastic metastases

Radiation‐induced morphea following irradiation of osteoblastic metastases

Prior elicitation of the efficacy and tolerability of Methotrexate and Mycophenolate Mofetil in Juvenile Localised Scleroderma

Diagnosis and management of morphoea in children: an overview

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