Successful autologous stem cell transplantation in two patients with juvenile dermatomyositis

Holzer, Ursula; Royen‐Kerkhof, Annet van; Torre, Patrick van der; Kuemmerle‐Deschner, J. B.; Well, C; Handgretinger, R.; Mueller, Ingo; Wulffraat, Nico

doi:10.3109/03009740903096622

Cited by 52 publications

(35 citation statements)

References 15 publications

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“…A short-term follow-up with detailed clinical information (including imaging before and after aSCT, and immune reconstitution after aSCT) of two patients describing complete remission was reported previously 4. The current follow-up of these patients is more than 5 years showing sustained remission.…”

mentioning

confidence: 76%

Autologous stem cell transplantation leads to a change in proinflammatory plasma cytokine profile of patients with juvenile dermatomyositis correlating with disease activity

et al. 2014

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mentioning

confidence: 76%

Autologous stem cell transplantation leads to a change in proinflammatory plasma cytokine profile of patients with juvenile dermatomyositis correlating with disease activity

et al. 2014

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“…A handful of case reports suggest that for severe, treatment refractory disease autologous HSCT can lead to sustained remission, although the response is not consistent [84,85]. However as with many of the CTDs, biological agents have meant that the role for autologous HSCT is limited to the most treatment-resistant of cases, so numbers are low.…”

Section: Other Connective Tissue Diseasesmentioning

confidence: 94%

Haematopoietic stem cell transplantation in autoimmune diseases: From basic science to clinical practice

Kelsey

Oliveira

Badoglio

et al. 2016

Current Research in Translational Medicine

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“…Autologous stem cell transplantation has been anecdotally reported to induce dramatic improvement and sustained remission in severe progressive JDM, SRP-or Jo-1-associated PM and adult DM patients unresponsive to conventional treatment [87,[90][91][92]. Nevertheless, less remarkable results have also been reported [93,94].…”

Section: Other Treatment Approachesmentioning

confidence: 95%

Idiopathic Inflammatory Myopathies: an Update on Classification and Treatment with Special Focus on Juvenile Forms

Pagnini

Vitale

Selmi

et al. 2015

Clinic Rev Allerg Immunol

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Juvenile inflammatory myopathies represent a heterogeneous group of rare and potentially fatal disorders of unknown aetiology, characterised by inflammation and proximal and symmetric muscle weakness. Beyond many similarities, specific clinical, laboratoristic and histopathologic features underlie different subsets with distinguishing demographic, prognostic and therapeutic peculiarities. Over time, several forms of inflammatory idiopathic myopathies have been described, including macrophagic myofascitis, immune-mediated necrozing myopathy and the spectrum of amyopathic dermatomyositis that include hypomyopathic dermatomyositis, inclusion body myositis and cancer-associated myositis occurring almost exclusively in adults. However, juvenile dermatomyositis is the most frequent in childhood, whereas polymyositis is relatively more frequent in adults. The aetiology is nowadays widely unclear; however, current theories contemplate a combination of environmental triggers, immune dysfunction and specific tissue responses involving muscle, skin and small vessels endothelium in genetically susceptible individuals. Myositis-specific autoantibodies, found almost exclusively in patients with myositis and myositis-associated autoantibodies, detectable both among patients with myositis and in subjects suffering from other autoimmune diseases, have an important clinical role because of their relation to specific clinical features, response to therapy and prognosis. The gold standard treatment for juvenile dermatomyositis is represented by corticosteroids, along with adjunctive steroid-sparing immunosuppressive therapies, which are used to counteract disease activity, prevent mortality, and reduce long-term disability. Further treatment approach such as biologic agents and autologous stem cell transplantation are emerging during the last years, in particular in patients difficult to treat and with poor prognosis. Therefore, a highly medical specialised approach is required for diagnosis and management of these conditions. This review comprehensively examines juvenile inflammatory myopathies focusing on clinical and laboratory classifications as well as on the current treatment approaches, referring in particular on biologic agents and latest therapeutic opportunities.

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Successful autologous stem cell transplantation in two patients with juvenile dermatomyositis

Cited by 52 publications

References 15 publications

Autologous stem cell transplantation leads to a change in proinflammatory plasma cytokine profile of patients with juvenile dermatomyositis correlating with disease activity

Autologous stem cell transplantation leads to a change in proinflammatory plasma cytokine profile of patients with juvenile dermatomyositis correlating with disease activity

Haematopoietic stem cell transplantation in autoimmune diseases: From basic science to clinical practice

Idiopathic Inflammatory Myopathies: an Update on Classification and Treatment with Special Focus on Juvenile Forms

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