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Keywords: fetal haemoglobin induction; -thalassaemia/HbE; pomalidomide; hydroxyurea; decitabine EN fetal haemoglobin induction -thalassaemia/HbE pomalidomide hydroxyurea decitabine e240 e245 6 06/18/20 20200615 NES 200615 Studies have shown that increased expression of fetal haemoglobin (HbF; SB 2 sb SB 2 sb ) can ameliorate red blood cell deficiencies in patients with -thalassaemia and sickle cell disease (SCD).[[1], [3]] Pharmacological induction of HbF expression in -thalassaemia has been investigated using several classes of small molecules,[4] including 5-azacytidine,[5] decitabine,[6] hydroxyurea,[7] LSD1 inhibitors (tranylcypromine and RN-1),[[8]] and short chain fatty acid derivatives.[[10]] Among these molecules, hydroxyurea is the only U.S. Food and Drug Administration (FDA) currently approved drug for the treatment of SCD and/or -thalassaemia. SP 0 sp -thalassaemia/HbE precursors from patients of different SP 0 sp -thalassemic mutations (Table SI) showed similarly increased levels of HbF induction in response to pomalidomide treatment. GLO:1XW/15jun20:bjh16670-fig-0002.jpg PHOTO (COLOR): 2 Effect of pomalidomide and its combinations on erythroid differentiation and mRNA expression of HbF regulators in cultured erythroid cells from 0-thalassaemia/HbE patients. HbF-inducing effects of hydroxyurea (HU), decitabine (DAC), and RN-1 in erythroid cells from 0-thalassaemia/HbE patients. [Extracted from the article]

We retrospectively analysed the outcomes of 127 children with acquired severe aplastic anaemia ( SAA) who had received haematopoietic stem cell transplantation ( HSCT) between 2000 and 2011 in one of the 10 Asia Pacific institutions. Fifty-three were matched sibling donor ( MSD) and 74 were alternative donor ( AD), including 22 matched unrelated donor, 32 mismatched unrelated donor and 20 mismatched related donor. With a median follow up 45·5 months (13-139) and when compared to the MSD group, AD recipients had more grade II- IV acute graft-versus-host disease (a GVHD; 14·3% vs. 32·8%, P = 0·029), but similar grade III- IV a GVHD (10·2% vs. 12·5%, P = 0·774), graft failure ( GF) (15·1% vs. 15·5%, P = 0·658) and 5-year overall survival (90·6% vs. 83·7%, P = 0·251). As a source of stem cell, peripheral blood stem cells ( PBSC) resulted in less GF (18% vs. 9·1% P = 0·013), similar grade II-IV a GVHD (28·1% vs. 17·4%, P = 0·258), chronic GVHD (25·8% vs. 29·3%, P = 0·822) and similar outcomes (89·7% vs. 82·4%, P =0 ·665) when compared to bone marrow ( BM). In univariate analysis, GF ( P < 0·001) and grade II- IV a GVHD ( P = 0·009) were predictors of poor survival. In multivariate analysis, only GF was associated with poor survival ( P = 0·012). The outcome of AD and PBSC HSCT were comparable to that of MSD and BM HSCT in the Asia Pacific region. [ABSTRACT FROM AUTHOR]