Diseases Potentially Treated by IUHSCTx
| (1) Diseases That May Benefit From IUHSCTx Alone Rationale = Selective advantage for donor cells SCID: X-Linked ZAP 70 Jac 3 ADA deficiency Wiskott Aldrich syndrome Chromosomal breakage syndromes: Fanconi anemia Bloom syndrome Rationale = Minimal engraftment requirement* Hyper IgM syndrome CGD |
| (2) Diseases That May Benefit From IUHSCTx in Combination With Minimally Ablative Postnatal Strategies Rationale = Successfully treated by mixed chimerism Hemoglobinopathies: β-Thalassemia/?α-thalassemia Sickle cell disease Rationale = Prenatal tolerance induction—No matched sibling donor Diseases that can be diagnosed early in gestation and successfully treated by postnatal SCT |
| (1) Diseases That May Benefit From IUHSCTx Alone Rationale = Selective advantage for donor cells SCID: X-Linked ZAP 70 Jac 3 ADA deficiency Wiskott Aldrich syndrome Chromosomal breakage syndromes: Fanconi anemia Bloom syndrome Rationale = Minimal engraftment requirement* Hyper IgM syndrome CGD |
| (2) Diseases That May Benefit From IUHSCTx in Combination With Minimally Ablative Postnatal Strategies Rationale = Successfully treated by mixed chimerism Hemoglobinopathies: β-Thalassemia/?α-thalassemia Sickle cell disease Rationale = Prenatal tolerance induction—No matched sibling donor Diseases that can be diagnosed early in gestation and successfully treated by postnatal SCT |
Could also be placed in category 2.