Treatment of Primary Immunodeficiency Diseases
Treatment of primary immunodeficiency diseases (PID) should be tailored to counteract the immune defect and prevent complications. Initiating treatment early in the course of the disease is essential. Prevention of infections is very important in all form
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Treatment of Primary Immunodeficiency Diseases
11
Hale Yarmohammadi and Charlotte Cunningham-Rundles
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Treatment of primary immunodeficiency diseases (PID) should be tailored to counteract the immune defect and prevent complications. Initiating treatment early in the course of the disease is essential. Prevention of infections is very important in all forms of PID. This can be achieved by reducing exposure to pathogens, with aggressive use of antimicrobial drugs, and with immunoglobulin substitution therapy. In infants with severe combined immunodeficiency, hematopoietic stem cell transplantation is the treatment of choice, and leads to permanent cure in the majority of patients. Replacement immunoglobulin is the mainstay of therapy for patients with predominantly antibody deficiencies. Autoimmune manifestations are not rare in PID. In general, their treatment should not differ from what is otherwise in use in immunocompetent individuals. Antibacterial and antifungal prophylaxis is essential in defects of phagocytosis. In addition, granulocyte-colony stimulating factor is the treatment of choice in patients with severe congenital neutropenia. Apart from combined immunodeficiencies, hematopoietic stem cell transplantation can be considered also in the treatment of severe phagocytes defects and of immunodeficiency with immune dysregulation.
11.1 Introduction The essential principles for therapy of patients with primary immunodeficiency diseases (PID) are to reduce exposure to infectious agents, aggressive use of
appropriate anti bacterial, antifungal and/or antiviral agents, and replacement or restoration of immunity where possible. An overview of different treatment modalities is shown in Table 11.1. About 70% of all patients with PID require immunoglobulin replacement, which its common indications are listed in Table 11.2. In this chapter, different options available for the treatment of some forms of PID are briefly discussed [15].
11.2 Therapy for Combined T and B Cell Immunodeficiencies 11.2.1 Severe Combined Immunodeficiency Once a diagnosis of severe combined immunodeficiency (SCID) is confirmed, therapy must be initiated as quickly as possible as it is a pediatric emergency. The first step is isolation, since infants with SCID are prone to fulminant life-threatening infections. The first line of therapy is antibiotics. Since Pneumocystis jiroveci pneumonia is a common early complication, infants should receive prophylaxis, usually trimethoprimsulfamethoxazole (5 mg/kg of trimethoprim given once daily 3 times a week). Alternative prophylactic regimens include pentamidine isethionate (5 mg/kg every 4 weeks), dapsone (1 mg/kg daily), and atovaquone (30 mg/kg daily). Early signs of infection should be promptly recognized, and antimicrobial regimens initiated early and for prolonged periods. Antiviral medications should be started when suspecting Varicella zoster virus (VZV) or cytomegalovirus (CMV) infections, which are common viral illnesses in this group. Since antibody
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