OBJECTIVES: The structural and functional changes of the hands and face in systemic sclerosis (SSc) can be severely disabling. We aimed to assess the effect of a 24-week supervised physiotherapy and occupational therapy program (POTp) combined with home exercise on the function of hands/mouth of SSc patients, compared to a daily home exercise program in typical outpatient care. METHODS: Fifty-nine patients with SSc were consecutively and non-selectively enrolled in an intervention (IG, n=27) or control (CG, n=32) group. Only the IG underwent the POTp twice a week for 1.5 hours. At baseline, 12, 24, and 48 weeks, all patients were assessed by a blinded physiotherapist for the hands/mouth function (delta finger-to-palm, handgrip strength, Hand and Mobility in Scleroderma, interincisal/interlabial distance), and self-evaluated their hand (Cochin Hand Function Scale) and mouth function (Mouth Handicap in Systemic Sclerosis scale), disability (Health Assessment Questionnaire [HAQ], SSc HAQ), and quality of life (Short Form-36). RESULTS: At week 24, compared to the significant deterioration in the CG, we found a significant improvement in the IG in the objectively assessed hands/mouth function and in the subjectively evaluated hand function and disability. The improvement was clinically meaningful (by >20%) in a substantial proportion of patients. Although the improvement in most outcomes was still present at week 48, the maximum effect was not sustained. CONCLUSIONS: This 24-week POTp not only attenuated the progressive deterioration, but also significantly improved the function of the hands/mouth, which was clinically meaningful in a substantial proportion of patients with SSc.
Musculoskeletal system impairment is a major cause of functional alterations in subjects with systemic sclerosis. Autologous hematopoietic stem cell therapy (AHSCT) may have an important role in the treatment functional of systemic sclerosis patients. The aim of this pilot study was to assess whether AHSCT interferes with the electromyographic activity of the masseter and temporalis muscles of subjects with systemic sclerosis. Before transplantation, seven subjects with systemic sclerosis (mean age [± SD], 40.1 ± 9.6 years) underwent electromyographic analysis of the masseter and temporalis muscles in mandibular tasks at rest, right and left laterality, protrusion and maximum voluntary contraction. Two months after AHSCT, the subjects re-evaluated using the same methods. Data were analyzed using the repeated-measure test, with p<0.05 considered to be statistically significant. Two months after AHSCT, there was reduction in normalized electromyographic activity in the dental clenching in maximal voluntary contraction, with significant differences, for the left temporal muscle (p=0.04). AHSCT in subjects with systemic sclerosis promotes alterations in stomatognathic system function, especially those related to electromyographic activity of masticatory muscles.
- Klíčová slova
- Electromyography, Hematopoietic stem cell transplantation, Masticatory muscles, Systemic sclerosis,
- MeSH
- dospělí MeSH
- elektromyografie MeSH
- lidé středního věku MeSH
- lidé MeSH
- pilotní projekty MeSH
- síla skusu * MeSH
- systémová sklerodermie * terapie MeSH
- transplantace hematopoetických kmenových buněk * MeSH
- žvýkací svaly MeSH
- Check Tag
- dospělí MeSH
- lidé středního věku MeSH
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
RATIONALE & OBJECTIVE: Data for outcomes of patients with end-stage renal disease (ESRD) secondary to systemic sclerosis (scleroderma) requiring renal replacement therapy (RRT) are limited. We examined the incidence and prevalence of ESRD due to scleroderma in Europe and the outcomes among these patients following initiation of RRT. STUDY DESIGN: Registry study of incidence and prevalence and a matched cohort study of clinical outcomes. SETTING & PARTICIPANTS: Patients represented in any of 19 renal registries that provided data to the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) Registry between 2002 and 2013. PREDICTOR: Scleroderma as the identified cause of ESRD. OUTCOMES: Incidence and prevalence of ESRD from scleroderma. Recovery from RRT dependence, patient survival after ESRD, and graft survival after kidney transplantation. ANALYTICAL APPROACH: Incidence and prevalence were calculated using population data from the European Union and standardized to population characteristics in 2005. Patient and graft survival were compared with 2 age- and sex-matched control groups without scleroderma: (1) diabetes mellitus as the cause of ESRD and (2) conditions other than diabetes mellitus as the cause of ESRD. Survival analyses were performed using Kaplan-Meier analysis and Cox regression. RESULTS: 342 patients with scleroderma (0.14% of all incident RRT patients) were included. Between 2002 and 2013, the range of adjusted annual incidence and prevalence rates of RRT for ESRD due to scleroderma were 0.11 to 0.26 and 0.73 to 0.95 per million population, respectively. Recovery of independent kidney function was greatest in the scleroderma group (7.6% vs 0.7% in diabetes mellitus and 2.0% in other primary kidney diseases control group patients, both P<0.001), though time required to achieve recovery was longer. The 5-year survival probability from day 91 of RRT among patients with scleroderma was 38.9% (95% CI, 32.0%-45.8%), whereas 5-year posttransplantation patient survival and 5-year allograft survival were 88.2% (95% CI, 75.3%-94.6%) and 72.4% (95% CI, 55.0%-84.0%), respectively. Adjusted mortality from day 91 on RRT was higher among patients with scleroderma than observed in both control groups (HRs of 1.25 [95% CI, 1.05-1.48] and 2.00 [95% CI, 1.69-2.39]). In contrast, patient and graft survival after kidney transplantation did not differ between patients with scleroderma and control groups. LIMITATIONS: No data for extrarenal manifestations, treatment, or recurrence. CONCLUSIONS: Survival of patients with scleroderma who receive dialysis for more than 90 days was worse than for those with other causes of ESRD. Patient survival after transplantation was similar to that observed among patients with ESRD due to other conditions. Patients with scleroderma had a higher rate of recovery from RRT dependence than controls.
- Klíčová slova
- Systemic sclerosis, dialysis, disease course, disease registry, end-stage renal disease (ESRD), incidence, kidney transplantation, mortality, outcomes, prognosis, renal recovery, renal replacement therapy (RRT), scleroderma,
- MeSH
- analýza přežití MeSH
- chronické selhání ledvin etiologie mortalita terapie MeSH
- dospělí MeSH
- hodnocení rizik MeSH
- internacionalita MeSH
- Kaplanův-Meierův odhad MeSH
- lidé středního věku MeSH
- lidé MeSH
- mladý dospělý MeSH
- náhrada funkce ledvin metody mortalita MeSH
- prediktivní hodnota testů MeSH
- příčina smrti * MeSH
- prognóza MeSH
- proporcionální rizikové modely MeSH
- registrace * MeSH
- retrospektivní studie MeSH
- senioři MeSH
- studie případů a kontrol MeSH
- systémová sklerodermie komplikace diagnóza terapie MeSH
- výsledek terapie MeSH
- Check Tag
- dospělí MeSH
- lidé středního věku MeSH
- lidé MeSH
- mladý dospělý MeSH
- mužské pohlaví MeSH
- senioři MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
- multicentrická studie MeSH
- práce podpořená grantem MeSH
- Geografické názvy
- Evropa MeSH
Systemic sclerosis is classed as a diffuse (systemic) disease of connective tissue. It is a heterogeneous disease significantly shortening life expectancy. Its etiology is unknown. Pathogenetic interplay is assumed to involve a triad of pathological autoimmune inflammation, vasculopathy and fibrosis. Clinical manifestations can be classed based on the preponderant pathogenetic process. Vasculopathy is manifested by secondary Raynauds phenomenon with abnormal findings on the nailfold capillaroscopy, skin telangiectasias, gastric antral vascular ectasia, life threatening scleroderma renal crisis, digital ulcerations and prognostically severe pulmonary arterial hypertension. The treatment of vascular manifestations uses medicines with vasodilation effect. The manifestation of inflammation is accentuated by pleurisy, pericarditis, myositis, synovitis/arthritis and alveolitis. Finally, the manifestation of fibrosis predominates in association with dermatosclerosis, interstitial lung disease and fibrotic impairment of the gastrointestinal tract. Medicines with immunomodulatory or immunosupressive effects are used to affect the inflammation and fibrosis. Despite the aforementioned, there is still no universally effective treatment available. The pharmacological therapy of this disease is organ specific and symptomatic.Key words: capillaroscopy - digital ulcers - interstitial lung disease - pulmonary arterial hypertension - scleroderma renal crisis - systemic sclerosis.
Systemic sclerosis (SSc) is a generalised connective tissue disease of unknown origin, which clinically shows by skin thickening and sclerosis of different extent (scleroderma) and by typical involvement of visceral organs. At the same time fibrotic and sclerotic changes occur in the blood vesel walls. SSc usually involves females at young and middle age. Myalgias, arthralgias and arthritis are nonspecific, tendon friction rubs in fingers are more typical for this diagnosis. Gastrointestinal involvement starts early in the oropharyngeal part, esophagus and proceeds into the distal parts. Fibrotic changes lead to slow transit dysmotility and pseudoobstruction and/or dilation of the bowels. The main symptoms are dysphagia, pyrosis, malabsorption and constipation. SSc produces two major patterns of abnormality within the lungs a fibrosing alveolitis or a primary pulmonary vascular disease. More frequently an insterstitial process develops which can be followed by pulmonary arterial hypertension. Cardiac involvement can also have different forms. Myocardial fibrosis usually appears at first in the conduction system by arrhythmias and various conduction blocks while pericarditis is mostly asymptomatic. Renal manifestation of SSc is observed in 8-10% patients. The most severe form--scleroderma renal crisis is characterised by the new onset of accelerated hypertension and rapidly progressive oliguric renal failure. No therapies have been proven to modify the course of SSc. Some of the drugs can affect only the skin changes. Majority of the currently applied agents have only a symptomatic effect.
Autoimmune diseases (AID) result from the impairment of the effector and/or recognition phase of the immune response. The autoimmune process plays a crucial role in the pathogenesis of the systemic lupus erythematosus (SLE), systemic sclerosis (SSc), rheumatoid arthritis (RA), and their treatment is therefore largely based on immunosuppression. However, some patients do not respond to its standard doses. The disease becomes intractable with the survival rate comparable to that of some haematological malignancies, or patients become soon handicapped with very poor quality of life, depending on continual administration of high doses of steroids. The new hope for those patients becomes therapy with high dose myelo- and immuno-ablative chemotherapy with autologous hematopoietic progenitor cell support (PBPC). Tens of patients with intractable forms of AID were transplanted in the pilot clinical studies with promising results. The most frequent indications included: SLE, SSc, and RA. Final conclusion of the therapeutic effects will be drawn from the analysis of larger trails.
- MeSH
- autoimunitní nemoci terapie MeSH
- imunosupresivní léčba MeSH
- lidé MeSH
- revmatoidní artritida terapie MeSH
- systémová sklerodermie terapie MeSH
- systémový lupus erythematodes terapie MeSH
- transplantace hematopoetických kmenových buněk * MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
- přehledy MeSH
- MeSH
- lidé středního věku MeSH
- lidé MeSH
- systémová sklerodermie diagnóza imunologie terapie MeSH
- termografie * MeSH
- transfer faktor terapeutické užití MeSH
- Check Tag
- lidé středního věku MeSH
- lidé MeSH
- ženské pohlaví MeSH
- Publikační typ
- anglický abstrakt MeSH
- časopisecké články MeSH
- Názvy látek
- transfer faktor MeSH
- Klíčová slova
- LUPUS ERYTHEMATOSUS/therapy *, SCLERODERMA/therapy *, VITAMIN E/therapeutic use *,
- MeSH
- alfa-tokoferol * MeSH
- Basidiomycota * MeSH
- lidé MeSH
- lokalizovaná sklerodermie * MeSH
- systémová sklerodermie terapie MeSH
- systémový lupus erythematodes terapie MeSH
- vitamin E terapeutické užití MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
- Názvy látek
- alfa-tokoferol * MeSH
- vitamin E MeSH
- Klíčová slova
- ACTH/therapeutic use *, SCLERODERMA/therapy *,
- MeSH
- adrenokortikotropní hormon terapeutické užití MeSH
- lokalizovaná sklerodermie * MeSH
- systémová sklerodermie terapie MeSH
- Publikační typ
- časopisecké články MeSH
- Názvy látek
- adrenokortikotropní hormon MeSH
