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10.1136/adc.65.4_spec_no.349 127

Articles with full access and available abstracts of paid publications were analyzed

In studies where 1000 mg was administered daily, equivalent to 20 capsules per day, only minimal effects were observed

We now recognize that, even for T-B + NK- SCID phenotypes, although full myeloid chimerism is not necessary for cure, a degree of myeloid engraftment will enable B-lymphocyte reconstitution and long-term thymopoiesis ( Key concepts for SCID: Newborn screening is now established in many countries Prevention of infection prior to definitive treatment affects outcome Conditioning prior to HSCT is recommended for all, to ensure good long-term outcomes Emerging IEI Patients with undefined T cell deficiencies historically had a worse outcome which maybe because transplant only took place as a last resort ( p = 0.006