Treatments Options by Type SCID: Early Treatment Saves Lives X-linked ADA-SCID Artemis SCID (aka SCID A or DCLRE1C) RAG-1 and RAG2 IL7R deficiency CD3 complex Component Deficiency CD45 deficiency Cernunnos-XLF Deficiency Coronin-1A deficiency DNA ligase 4 deficiency DNA-PKcs deficiency JAK3 deficiency LAT deficiency Reticular dysgenesis Leaky SCID Omenn Syndrome Early diagnosis and treatment for SCID is necessary for improving a child’s chances of survival. If a child is diagnosed and treated within the first few months of life before a serious infection develops, then the long-term survival rate is more than 90%. With early treatment, most children with SCID should be able to develop their own working immune system. The best course of treatment for a child with SCID depends on several factors including the type of SCID and the child’s health. Hematopoietic Stem Cell Transplant (HSCT) is the standard treatment for all types of SCID. In HSCT, donor stem cells are introduced into the child and develop an immune system. Enzyme Replacement Therapy is a temporary treatment that is used to treat ADA-SCID. Gene therapy is an alternative treatment in clinical trials available only for X-linked SCID, ADA-SCID, and Artemis SCID. Gene therapy uses a child's own corrected stem cells to build an immune system. SCID Types SCID Diagnosis This project is supported by the Health Resources and Services Administration (HRSA) of the U.S. Department of Health and Human Services (HHS) as part of an award totaling $4 million with 0% finances with nongovernmental sources. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement, by HRSA, HHS or the U.S. Government.

SCID: Early Treat m ent Saves Lives - primaryimmune.org › sites › default › files › SCID_ Treatment a… · SCID: Early Treat m ent Saves Lives X-linked ADA-SCID Artemis SCID

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Treatments Options by Type

SCID: Early Treatment Saves Lives

X-linkedADA-SCIDArtemis SCID (aka SCIDA or DCLRE1C)RAG-1 and RAG2IL7R deficiencyCD3 complexComponent DeficiencyCD45 deficiencyCernunnos-XLFDeficiencyCoronin-1A deficiencyDNA ligase 4 deficiencyDNA-PKcs deficiencyJAK3 deficiencyLAT deficiencyReticular dysgenesisLeaky SCIDOmenn Syndrome

Early diagnosis and treatment for SCID is necessary forimproving a child’s chances of survival. If a child isdiagnosed and treated within the first few months of lifebefore a serious infection develops, then the long-termsurvival rate is more than 90%. With early treatment,most children with SCID should be able to develop theirown working immune system. The best course oftreatment for a child with SCID depends on severalfactors including the type of SCID and the child’s health.

Hematopoietic Stem Cell Transplant (HSCT) is the standardtreatment for all types of SCID. In HSCT, donor stem cells areintroduced into the child and develop an immune system. Enzyme Replacement Therapy is a temporary treatment that isused to treat ADA-SCID. Gene therapy is an alternative treatment in clinical trialsavailable only for X-linked SCID, ADA-SCID, and Artemis SCID.Gene therapy uses a child's own corrected stem cells to buildan immune system.

SCID TypesSCID Diagnosis

This project is supported by the HealthResources and Services Administration(HRSA) of the U.S. Department of Healthand Human Services (HHS) as part of anaward totaling $4 million with 0% financeswith nongovernmental sources. Thecontents are those of the author(s) and donot necessarily represent the official viewsof, nor an endorsement, by HRSA, HHS orthe U.S. Government.