Immunologic targets for currently available immunosuppressive agents: What is the optimal approach for children?

SummaryInhibitor development is a rare but serious event in hemophilia B patients. Management is hampered by the frequent occurrence of allergic reactions to factor IX, low success rates of current inhibitor elimination protocols and the risk of development of nephrotic syndrome. Single cases of immune tolerance induction (ITI) including immunosuppressive agents like mycophenolat mofetil (MMF) or rituximab have been reported. We present a case of successful inhibitor elimination with a combined immune-modulating therapy and high-dose factor IX (FIX). This boy had developed a FIX inhibitor at the age of 5 years and had a history of allergic reactions to FIX and to FEIBA→. Under on-demand treatment with recombinant activated FVII the inhibitor became undetectable but the boy suffered from multiple joint and muscle bleeds. At the age of 11.5 years ITI was attempted with a combination of rituximab, MMF, dexamethasone, intravenous immunoglobulins and high-dose FIX. The inhibitor did not reappear and FIX half-life normalized. No allergic reaction, no signs of nephrotic syndrome and no serious infections were observed.

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Overview of Biological Therapy in Ulcerative Colitis: Current and Future Directions

Journal of Gastrointestinal and Liver Diseases ◽

10.15403/jgld.2014.1121.242.bezz ◽

2015 ◽

Vol 24 (2) ◽

pp. 203-213 ◽

Cited By ~ 9

Author(s):

Federica Furfaro ◽

Cristina Bezzio ◽

Sandro Ardizzone ◽

Alessandro Massari ◽

Roberto De Franchis ◽

...

Keyword(s):

Ulcerative Colitis ◽

Immunosuppressive Agents ◽

Mucosal Healing ◽

Aminosalicylic Acid ◽

Proinflammatory Mediators ◽

Experimental Drugs ◽

New Treatments ◽

Aggressive Clinical Course ◽

Near Future

The treatment of ulcerative colitis (UC) has changed over the last decade. It is extremely important to optimize the therapies which are available nowadays and commonly used in daily clinical practice, as well as to stimulate the search for more powerful drugs for the induction and maintenance of sustained and durable remission, thus preventing further complications. Therefore, it is mandatory to identify the patients' prognostic variables associated with an aggressive clinical course and to test the most potent therapies accordingly.To date, the conventional therapeutic approach based on corticosteroids, salicylates (sulfasalazine, 5-aminosalicylic acid) or immunosuppressive agents is commonly used as a first step to induce and to maintain remission. However, in recent years, knowledge of new pathogenetic mechanisms of ulcerative colitis have allowed us to find new therapeutic targets leading to the development of new treatments that directly target proinflammatory mediators, such as TNF-alpha, cytokines, membrane migration agents, cellular therapies.The aim of this review is to provide the most significant data regarding the therapeutic role of drugs in UC and to give an overview of biological and experimental drugs that will become available in the near future. In particular, we will analyse the role of these drugs in the treatment of acute flare and maintenance of UC, as well as its importance in mucosal healing and in treating patients at a high risk of relapse.

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New therapeutic approaches for polymyositis and dermatomyositis

Orvosi Hetilap ◽

10.1556/oh.2011.29176 ◽

2011 ◽

Vol 152 (39) ◽

pp. 1552-1559 ◽

Cited By ~ 2

Author(s):

Katalin Dankó ◽

Melinda Vincze

Keyword(s):

Immunosuppressive Agents ◽

Inflammatory Myopathy ◽

Proximal Muscle ◽

Therapeutic Approaches ◽

First Line ◽

New Therapeutic Approaches ◽

Remission Period ◽

Immune Mediated ◽

Systemic Manifestations ◽

Line Of Therapy

Inflammatory myopathies are chronic, immune-mediated diseases characterized with progressive proximal muscle weakness. They encompass a variety of syndromes with protean manifestations. The aims of therapy are to increase muscle strength, prevent the development of contractures, and to manage the systemic manifestations of the disease. This is a complex treatment which requires routine and wide knowledge. The most important task is to recognize the disease and guide the patient to immunologic center. Although the first line of therapy continues to include corticosteroids, there are a multitude of agents available for treating patients with myositis. There are several different immunosuppressive agents which may be applied alone or in combination with each other, as well as an increasing number of novel and exciting biologic agents targeting molecules participating in the pathogenesis of inflammatory myopathy. Physiotherapy and rehabilitation in the remission period may significantly improve the functional outcome of patients with these disorders. Orv. Hetil., 2011, 152, 1552–1559.

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