Key clinical point: An alemtuzumab/fludarabine conditioning regimen, without any radiation or alkylating agents, was effective in patients with dyskeratosis congenita who experienced myeloid failure.
Major finding: In total, 95% of patients with dyskeratosis congenita achieved primary engraftment at a median of 22 days after transplant.
Study details: A prospective, multicenter study including 20 patients with dyskeratosis congenita aged from 30 months to 65 years.
Disclosures: Dr. Agarwal reported having no conflicts of interest.
Agarwal S et al. TCT 2019, Abstract LBA2.
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