HSCT and genetic conditions

by Yannis Trakadis, MD

Posted February 28th, 2016 by Yannis Trakadis & filed under Treatment.

Hematopoietic stem cell transplantation (HSCT) with matched unrelated donors can be used in different non-malignant genetic diseases. Abdel-Azim et al. report the outcomes of 15 patients with non-malignant genetic diseases who received HSCT from 2006 to 2014.

Fifteen patients were enrolled during the study period: 26.7% female (n = 4), 33.3% Hispanic (n = 5), 40% African American (n = 6), 26.7% Caucasian (n = 4).

Median patient age at transplant was 7 (0.8–17.6) years. Diagnoses included HLH (n = 2), CD40 ligand deficiency [CD40-L] (n = 2), congenital dyserythropoietic anemia [CDA] type I (n = 1), CDA type II (n = 1), ALD (n = 1), sickle cell diseases [SCD] (N = 7), and b-thalassemia (n = 1, class II).

At a median follow-up of 2 (0.2-5.4) years, the overall survival (OS) was 93.3% (95% CI: 0.61-0.99) and disease-free survival (DFS) was 73.3% (95% CI: 0.44-0.89).

Alemtuzumab was associated with durable donor engraftment and relatively low rates of regimen related toxicity.

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Unrelated donor hematopoietic stem cell transplantation for the treatment of non-malignant genetic diseases: An alemtuzumab based regimen is associated with cure of clinical disease; earlier clearance of alemtuzumab may be associated with graft rejection. Abdel-Azim  H, Mahadeo  KM, Zhao  Q, Khazal  S, Kohn  DB, Crooks  GM, Shah  AJ, Kapoor  N. Am J Hematol. 2015 Nov;90(11):1021-–6. PMID:
[PubMed: 26242764]

Posted by Yannis Trakadis, MD