112. Thalassemia and Globin Gene Regulation: Clinical Advances in Thalassemia

Monday, December 5, 2016: 2:45 PM-4:15 PM
Ann Dean, PhD, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health and Phoebe Joy Ho, MBBS DPhil FRACP FRCPA, Royal Prince Alfred Hospital

Qiao chuan, Jian ming Luo, Zhong ming Zhang, Lian jin Liu, et al.

Conclusion: Our results demonstrate that grafts composed of PB + BM had superior overall outcomes compared to CB + BM grafts, as evidenced by faster engraftment and lower TRM of the former despite substantially lower aGVHD and cGVHD rates of the latter. The mixed stem cell populaitons and the high cell dose achieved with the use of 2 different graft sources, toghether with the conditioning regimen used likely contributed to the superior outcomes seen with this regiem. This strategy could be of great benefit for the treatment of patient with TM and other benign hematologic disease.

Chunfu Li, Yuelin He, Xuedong Wu, Xiaoqin Feng, et al.

SummaryCT-hap-CB resulted in high OS and TFS, especially CT-14 leaded to 100% TFS with low GVHD rate. MSC engraftment improved hematopoietic recovery of UCB. KIR and HLA typing impacted what stem cells engraftment. The multicenter study should be developed in the future.


Alessia Pepe, Silvia Maffei, Laura Pistoia, Angela Ciancio, et al.

Conclusion. Females seem to tolerate iron toxicity better, possibly as an effect of reduced sensitivity to chronic oxidative stress. According to the International Guidelines, TM patients should perform a complete cardiac evaluation every year. Our study suggested that in females older than 20 years the follow-up may be performed every 24 months, thus reducing health care costs.


Ali T. Taher, Raffaella Origa, Silverio Perrotta, Alexandra Kouraklis, et al.

Conclusions: These results show a clear preference in favor of DFX FCT in all domains for the modified SICT. Pts were satisfied with their medicine during the study period and more pts were satisfied with DFX FCT compared with DT at all visits. DFX FCT offers pts an improved formulation that does not require administration in a fasting state, has better palatability, and very little concern associated with GI tolerability. Enhanced pt satisfaction with the new DFX FCT formulation may improve adherence, thereby, reducing iron overload-related complications.


Antonio G. Piga, Immacolata Tartaglione, Rita Gamberini, Ersi Voskaridou, et al.

Conclusions: Luspatercept treatment in pts with beta-thalassemia had a favorable safety profile. Efficacy was clinically relevant in both NTD pts (increased Hgb levels, decreased LIC, and improved quality of life) and TD pts (decreased RBC transfusions). A Phase 3, double-blind, placebo-controlled study of luspatercept in regularly transfused adults with beta-thalassemia is ongoing (NCT02604433).


Yesim Aydinok, Zeynep Karakas, Elena Cassinerio, Noppadol Siritanaratkul, et al.

CONCLUSION: RUX Tx showed a trend for improvement in transfused red cells and a slight improvement in pre-transfusion Hgb; while, there was a noticeable reduction in SV over time. As per investigator assessment of clinical benefit, a majority of pts continued Tx beyond the core study. RUX was well tolerated in the study population with modest incidences of G 3 or 4 and serious AEs, with no new safety findings. Given the sustained decrease in SV, further studies could be valuable to determine if RUX Tx may be an alternative to splenectomy in pts with TDT.