Combined B-Cell and T-Cell Disorders Medication

  • Author: Francisco J Hernandez-Ilizaliturri, MD; Chief Editor: Emmanuel C Besa, MD   more...
 
Updated: Nov 28, 2011
 

Medication Summary

The goals of pharmacotherapy are to reduce morbidity and to prevent complications.

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Blood Products/Immunoglobulins

Class Summary

Blood products/immunoglobulins provide immediate passive immunity. These agents can be used as replacement therapy in patients with antibody-deficiency states.

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Immune globulin, intravenous (Gamimune, Gammagard, Sandoglobulin, Gammar-P)

 

Provide an immediate rise of antibodies that have a proven protective effect against bacterial and viral infection (passive immunity). Because antibodies are not produced by the host, these products must be readministered monthly. This treatment may increase CSF IgG (10%).

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Metabolic Enzymes

Class Summary

Metabolic enzymes are used to replace ADA.

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Pegademase bovine (Adagen)

 

ADA is an enzyme of the purine salvage pathway that is responsible for adenosine and deoxyadenosine deamination to inosine and deoxyinosine, respectively. ADA deficiency leads to accumulation of the metabolites dATP and 2'-deoxyadenosine, both of which are toxic to lymphocytes.

Treatment is indicated in patients with SCID secondary to ADA deficiency whose conditions proved refractory to bone marrow transplantation or who are not candidates for transplantation. Individualize therapy (based on plasma levels) to achieve the following: trough plasma levels of 15-35 mmol/h/mL and a decline in erythrocyte dATP to < 0.005-0.015 mmol/mL packed erythrocytes or to < 1% of total erythrocyte adenine nucleotide content (ATP + dATP). Plasma levels >35 mmol/h/mL are not associated with additional clinical benefit. This treatment has no role in preparatory regimen for bone marrow transplantation.

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Contributor Information and Disclosures
Author

Francisco J Hernandez-Ilizaliturri, MD  Assistant Professor, Departments of Medicine and Immunology, Roswell Park Cancer Institute, University of Buffalo State University of New York School of Medicine and Biomedical Sciences

Francisco J Hernandez-Ilizaliturri, MD is a member of the following medical societies: American Association for Cancer Research and American Society of Hematology

Disclosure: Nothing to disclose.

Coauthor(s)

Mohammad Muhsin Chisti, MD  Fellow in Hematology and Oncology, Beaumont Cancer Center, Oakland University William Beaumont School of Medicine

Mohammad Muhsin Chisti, MD is a member of the following medical societies: American College of Physicians, American Medical Association, American Society of Clinical Oncology, American Society of Hematology, and Medical Society of the State of New York

Disclosure: Nothing to disclose.

Issam Makhoul, MD  Associate Professor, Department of Medicine, Division of Hematology/Oncology, University of Arkansas for Medical Sciences

Issam Makhoul, MD is a member of the following medical societies: American Society of Clinical Oncology and American Society of Hematology

Disclosure: Nothing to disclose.

David Claxton, MD  Professor of Medicine, Department of Internal Medicine, Section of Hematology-Oncology, Hershey Medical Center, Pennsylvania State University College of Medicine

Disclosure: Nothing to disclose.

James O Ballard, MD  Kienle Chair for Humane Medicine, Professor, Departments of Humanities, Medicine, and Pathology, Division of Hematology/Oncology, Milton S Hershey Medical Center, Pennsylvania State University College of Medicine

James O Ballard, MD is a member of the following medical societies: Alpha Omega Alpha, American College of Physicians, and American Society of Hematology

Disclosure: Nothing to disclose.

Specialty Editor Board

Charles S Greenberg, MD  Director of Thrombosis and Transglutaminase Research Laboratory, Professor, Departments of Pathology and Medicine, Division of Hematology/Oncology, Duke University Medical Center

Charles S Greenberg, MD is a member of the following medical societies: American Society of Hematology and International Society on Thrombosis and Haemostasis

Disclosure: Nothing to disclose.

Francisco Talavera, PharmD, PhD  Adjunct Assistant Professor, University of Nebraska Medical Center College of Pharmacy; Editor-in-Chief, Medscape Drug Reference

Disclosure: Medscape Salary Employment

Marcel E Conrad, MD  Distinguished Professor of Medicine (Retired), University of South Alabama College of Medicine

Marcel E Conrad, MD is a member of the following medical societies: Alpha Omega Alpha, American Association for the Advancement of Science, American Association of Blood Banks, American Chemical Society, American College of Physicians, American Physiological Society, American Society for Clinical Investigation, American Society of Hematology, Association of American Physicians, Association of Military Surgeons of the US, International Society of Hematology, Society for Experimental Biology and Medicine, and Southwest Oncology Group

Disclosure: No financial interests None None

Rajalaxmi McKenna, MD, FACP  Southwest Medical Consultants, SC, Department of Medicine, Good Samaritan Hospital, Advocate Health Systems

Rajalaxmi McKenna, MD, FACP is a member of the following medical societies: American Society of Clinical Oncology, American Society of Hematology, and International Society on Thrombosis and Haemostasis

Disclosure: Nothing to disclose.

Chief Editor

Emmanuel C Besa, MD  Professor, Department of Medicine, Division of Hematologic Malignancies, Kimmel Cancer Center, Jefferson Medical College of Thomas Jefferson University

Emmanuel C Besa, MD is a member of the following medical societies: American Association for Cancer Education, American College of Clinical Pharmacology, American Federation for Medical Research, American Society of Clinical Oncology, American Society of Hematology, and New York Academy of Sciences

Disclosure: Nothing to disclose.

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Table 1. Classification of SCID
PathophysiologyCells AffectedInheritanceGenes Involved
Premature cell deathT, B, NKARADA
Defective cytokine–dependent survival signalingT, NKAR



γ c type-XL



JAK3, IL7RA (T cells only), γ c
Defective V(D)J rearrangementT, BARRAG1, RAG2, Artemis
Defective pre-TCR and TCR signalingTARCD3 δ, CD3 ζ, CD3 ε,



CD45



AR = autosomal recessive; JAK3 =Janus tyrosine kinase 3; RAG1, RAG2 = recombinase activating gene 1 and 2, respectively; TCR = T-cell receptor; XL = X-linked; V(D)J = variable diversity joining.
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