The main purpose of this study is to assess the effects of
Dyskeratosis congenita (DC) is an inherited multisystem disorder, which classically presents with a clinical triad of skin pigment abnormalities, nail dystrophy, and oral leukoplakia. DC is part of a spectrum of
The purpose of this
This will be an open-label, single arm, phase IIa study in subjects with AA or Hypoplastic MDS.
The objective of this trial is to make an initial assessment for this new treatment regimen and to show it is equal or superior to the current standard practice. With this initial assessment be hope to gain information suggesting further study of this regimen or discontinuation of this regimen before exposing large numbers of patients to this new treatment option. We also will gain experience with this new regimen giving insights as to possible modifications in dosing and monitoring and selection of patients for future treatment in case of positive results.
Allogeneic hematopoietic stem cell transplantation (aHSCT) can cure patients with a variety of