Old medications and new targeted therapies in systemic sclerosis

Nagaraja, Vivek; Denton, Christopher P.; Khanna, Dinesh

doi:10.1093/rheumatology/keu285

Cited by 49 publications

(57 citation statements)

References 77 publications

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“…1 Although there has been significant progress over the years in identifying the early phases of the disease, and recognizing different clinical phenotypes with variable course, really effective therapy remains an unsolved issue. 2 The pathogenesis of SSc is still poorly understood, hence treatment of this condition is difficult and often 'organ-based'. Treatment options have targeted different pathogenic processes, including inflammation, immune dysregulation and fibrosis, showing only limited efficacy.…”

Section: Introductionmentioning

confidence: 99%

Autologous hematopoietic stem cell transplantation has better outcomes than conventional therapies in patients with rapidly progressive systemic sclerosis

Papa

Onida

Zaccara

et al. 2016

Bone Marrow Transplant

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We retrospectively evaluated the efficacy of autologous hematopoietic stem cell transplantation (AHSCT) in 18 patients with rapidly progressive diffuse cutaneous systemic sclerosis (rp-dcSSc), and compared their disease outcomes with those of 36 demographically-and clinically-matched patients treated with conventional therapies. Cutaneous involvement, by performing modified Rodnan skin score (mRss), lung diffusion capacity, by measuring diffusing capacity of lung for carbon monoxide (DLCO), and disease activity, by applying the European Scleroderma Study Group (ESSG) scoring system, were the outcome variables measured at the baseline time and then every 12 months for the following 60 months in both the AHSCT-treated patients and the control group. In the AHSCT group, treatment-related mortality was 5.6%. In this group, both mRss and ESSG scores showed a significant reduction 1 year after AHSCT (P o 0.002); and these results were maintained until the end of follow-up. Conversely, DLCO values remained stable during the whole period of follow-up. Survival rate of AHSCT group was much higher than that observed in the whole control group (P = 0.0005). The probability that the ESSG score and mRss would remain at a high level, and DLCO could decrease, was significantly higher in the control group as a whole and in the subgroup of control patients treated with cyclophosphamide than in the AHSCT group. This study confirms that the AHSCT is effective in prolonging survival, as well as in inducing a rapid reduction of skin involvement and disease activity, and preserving lung function in patients with rp-dcSSc.

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Section: Introductionmentioning

confidence: 99%

Autologous hematopoietic stem cell transplantation has better outcomes than conventional therapies in patients with rapidly progressive systemic sclerosis

Papa

Onida

Zaccara

et al. 2016

Bone Marrow Transplant

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“…There have been no clinical trials on the isolated use of CYC in the therapy of skin induration. An analysis of results obtained in Scleroderma Lung Study-I, including 85 patients with dcSSc who were treated with CYC for 12 months, has shown a significant improvement and differences in modified Rodnan Skin Score (mRSS) compared to placebo [24]. There have been no randomized ból w klatce piersiowej, obrzęk śluzówek nosa oraz niedokrwistość [17].…”

Section: Skin Involvementmentioning

confidence: 99%

“…A prolowanego stosowania CYC w terapii stwardnień skóry. Przeprowadzona analiza wyników badania 85 chorych z dcSSc Scleroderma Lung Study-I, którzy otrzymywali CYC przez 12 miesięcy, wykazała znaczącą poprawę i różnice w modified Rodnan Skin Score (mRSS) w porów-naniu z placebo [24]. Nie ma randomizowanych badań klinicznych oceniających skuteczność MMF w zakresie redukcji nasilenia zmian skórnych ocenianych za pomocą mRSS, ale badania otwarte i retrospektywne wskazują, że MMF zmniejszał stwardnienia skóry w przebiegu dcSSc [25].…”

Section: Scleroderma Renal Crisisunclassified

Systemic sclerosis – diagnostic and therapeutic recommendations of the Polish Dermatological Society. Part 2: treatment

Krasowska¹,

Rudnicka²,

Dańczak‐Pazdrowska³

et al. 2017

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Systemic sclerosis is an immune-mediated disease characterized by a chronic progressive course. The complex and diverse clinical features seen in patients require case-by-case approach and cooperation of multiple specialists both at the stage of diagnosis and treatment. Critical factors in systemic sclerosis include early diagnosis, assessment of internal organ involvement, identification of patients at potential risk of organ complications, assessment of disease dynamics and activity, and implementation of optimal therapy. Treatment decisions should be made individually for each patient after careful consideration of such aspects as the severity of skin lesions, duration and activity of the disease, manifestations and possible internal organ involvement. On account of potential organ dysfunctions, patients require a multidisciplinary therapeutic approach and coordinated treatment by experienced specialists or in specialist medical centres. Part 2 presents current guidelines for the treatment of systemic sclerosis. pracy wielospecjalistycznej zarówno na etapie diagnozowania, jak i leczenia. W twardzinie układowej ważne są wczesne rozpoznanie, ocena zajęcia procesem chorobowym narządów wewnętrznych, identyfikacja chorych z ryzykiem wystąpienia powikłań narządowych, ocena dynamiki i aktywności choroby i wdrożenie optymalnej terapii. Decyzja o leczeniu powinna być podejmowana indywidualnie dla każdego pacjenta po wnikliwej analizie nasilenia zmian skórnych, czasu trwania, oceny aktywności choroby oraz występujących dolegliwości i zmian w narządach wewnętrznych. Ze względu na potencjalne dysfunkcje narządowe pacjent wymaga wielodyscyplinarnego podejścia terapeutycznego i koordynowania leczenia przez doświadczonych specjalistów lub w ośrodkach specjalistycznych. W części drugiej przedstawiono aktualne rekomendacje dotyczące leczenia.

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“…Unfortunately, controlled studies analyzing the effects of different maintenance therapies such as azathioprine or mycophenolate mophetil (MMF) after CYC treatment are not available. In addition, the optimal treatment duration with CYC is not known [77].…”

Section: Cyclophosphamidementioning

confidence: 99%

“…Three controlled trials on HSCT in SSc have been published so far [77]. The Autologous Stem Cell Transplantation International Scleroderma (ASTIS) Trial revealed that HSCT-treated patients had significant better long-term event-free survival (defined as death or major organ failure), with the cost of a higher treatment-related mortality in the first year after therapy.…”

Section: Other Emerging Treatments-biologics and Stem Cell Transplantmentioning

confidence: 99%

Management of Scleroderma-Associated Pulmonary Involvement

Dobrotă

Distler

Wells

et al. 2015

Curr Treat Options in Rheum

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Old medications and new targeted therapies in systemic sclerosis

Cited by 49 publications

References 77 publications

Autologous hematopoietic stem cell transplantation has better outcomes than conventional therapies in patients with rapidly progressive systemic sclerosis

Autologous hematopoietic stem cell transplantation has better outcomes than conventional therapies in patients with rapidly progressive systemic sclerosis

Systemic sclerosis – diagnostic and therapeutic recommendations of the Polish Dermatological Society. Part 2: treatment

Management of Scleroderma-Associated Pulmonary Involvement

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