Stem cell therapies for systemic sclerosis

The presence of autoimmune diseases, including Systemic Sclerosis (SSc), suggest failure of the normal immune regulatory processes leading to activation and expansion of autoreactive effector immune cells. Recently, stem cell transplantation emerged as a novel rescue therapy for a variety of refractory autoimmune diseases. The therapeutic strategy involves the ablation of the aberrant self‐reactive immune cells by chemotherapy and the regeneration of a new self‐tolerant immune system formed by the transplanted stem cells. In the last few years, thousands of patients worldwide have received haematopoietic stem cell transplantation (HSCT), mostly autologous, as treatment for severe irreversible autoimmune diseases, with promising results. Here we review the results of published small series of SSc patients treated with allogeneic and autologous HSCT, as well as three randomized trials, exploring the safety and efficacy of autologous HSCT in SSc. Although the results are encouraging, nonetheless, the correct application of stem cell transplantation remains an area of active investigation. Results of larger randomized, double blind clinical trials, will certainly improve our knowledge of the appropriate clinical use of stem cell therapy in SSc patients.     

Prognostic factors of macrophage activation syndrome,at the time of diagnosis,in adult patients affected by autoimmune disease: Analysis of 41 cases collected in 2 rheumatologic centers

Macrophage activation syndrome (MAS) is a rare, life-threatening disease in which early diagnosis and aggressive therapeutic strategy may improve the outcome. Due to its rarity, epidemiologic data are still lacking. Hyperferritinemia is frequently associated with MAS and might modulate the cytokine storm, which is involved in the development of multiple organ failure.In this paper, we investigated clinical data, treatments, and outcome of a homogeneous cohort of 41 adult MAS patients, complicating autoimmune rheumatic diseases. MAS-related death occurred in 17 patients (42.5%) during the follow-up, and older age and increased serum ferritin levels, at the time of diagnosis, were significantly associated with mortality.In conclusion, adult MAS is associated with high mortality rate. Some clinical features at diagnosis may be predictive of MAS-associated death.     

Glucocorticoids in rheumatoid arthritis: the silent companion in the therapeutic strategy

ABSTRACT

Introduction

Glucocorticoids (GCs) are key actors in RA management, despite the increasing number of available drugs. In fact, due to their efficacy and safety, the combination therapy between GCs and conventional synthetic disease-modifying anti-rheumatic drugs (csDMARDs) is still recommended in the early phase of RA treatment, because improving the long-term results.     

Cerebral abscesses: topic treatment with antibiotics

The Authors, after a brief discussion of current therapies used in the treatment of cerebral abscesses, present a series of 10 cases treated topically with antibiotics (through drill-hole and puncturing of the abscess). The determining role of CT scan in this technique is emphasised.     

Tocilizumab for the treatment of adult-onset Still’s disease: results from a case series

Adult-onset Still’s disease (AOSD) is a chronic inflammatory disease of unknown etiology which commonly affects young adults. Treatment of AOSD patients includes nonsteroidal anti-inflammatory drugs, corticosteroids, and DMARDs. Interleukin (IL)-6 blockade is an attractive therapeutic option for AOSD because this cytokine contributes to the pathogenesis of major AOSD symptoms. Tocilizumab (TCZ) is a humanized anti-IL-6 receptor antibody that blocks the effects of IL-6. Preliminary results of TCZ in AOSD have been promising. Here, we reported our experience evaluating both the safety and the efficacy of 12 months therapy with TCZ in 11 patients with AOSD refractory to corticosteroids and MTX therapy, followed for 18 months, including the first 12 months of active treatment and the last 6 months to evaluate the activity of the disease when the treatment was discontinued. The main outcome measures were the European League Against Rheumatism (EULAR) improvement criteria and improvement of systemic symptoms at the 3, 6, 12, and 18-months follow-up periods. Our patients rapidly responded and experienced a sustained clinical remission over time during active treatment. Disease Activity Score 28 decreased from 5.62 (3.75–8.28) [median (range)] at baseline to 1.61(0.49–3.5) at month 12. EULAR remission was achieved in 81.82 % at 12 months. Tender joint and swollen joint counts displayed a progressive reduction during active therapy study period. During treatment, we observed a resolution of fever in our AOSD patient. In conclusion, TCZ might represent a suitable option for the therapy of refractory AOSD patients.     

Ambulatory electrocardiographic monitoring in myotonic dystrophy (Steinert's Disease). A study of 22 patients

Ambulatory electrocardiographic monitoring (AEM) was performed in 22 patients (range 13-62 years; mean age 38.2 +/- 12.7) with grades I, II and III of myotonic dystrophy in order to evaluate the occurrence of potentially dangerous cardiac arrhythmias and conduction disturbances. All patients had previously undergone echocardiographic examination to determine whether structure and function abnormalities were present. In 6 patients with normal resting electrocardiogram, AEM revealed: first degree A-V block (4 cases), class IVa Lown ventricular arrhythmias (3 cases) and episodes of atrial fibrillation (4 cases). In 2 of 3 cases with abnormal scalar electrocardiogram new abnormalities (first degree A-V block and further prolongation of P-R interval) were demonstrated by AEM. Only 1 patient had mild signs of left ventricular dysfunction at echo. Disorders of cardiac conduction and rhythm are characteristic of myotonic dystrophy and can predispose to severe cardiac events. In this respect AEM is shown to be an early and sensitive tool in identifying patients at risk.