[AAteen]

I have not posted here for long but visited this forum from time to time. My prayers are with you all who suffer or have loved ones suffering from this disease.

My teenage niece was diagnosed moderate AA in early 2009 and treated ATG in mid-2010 with limited response (pre ATG: Hgb 7 – 8, ANC: 0.8 – 1, Plt 15k to 20k). Initial response of RBC and ANC to the ATG was quite good, but declined soon. Platelet rose very slowly, reached 48k highest at about 6 months after ATG, but then declined to 20k - 30k, then 14k - 20k. Seems ATG had initial effect but cannot sustain. My niece was RBC transfusion free for one year until early 2012. In early 2013, she got serious infection and required platelet transfusion for the 1st time (total 4 times in 2 months). She then resumed cyclosporine alone in March 2013, and then platelet and Hgb rose to just above transfusion threshold until early this year when she required RBC transfusion again. Currently still on cyclosporine (150 mg twice per day)

The recent BMB in Jan 2014 revealed chromosome abnormality of trisomy 8. There was no cytogenetic abnormality before ATG and this is the first cytogenetic study after ATG. We wondered whether the cytogenetic abnormality is caused by the ATG treatment or the cyclosporine. Doctor worry that it will progress to MDS and AML and recommend BMT, even no sibling match. Best unrelated donor available is only 7/8 match with mismatch at HLA- B allele. We in Hong Kong only conduct HLA typing for HLA-A, B, C and DRB1. Doctors seem not concerned about the mismatch. But as there is no 8/8 match identified, we are very hesitant. I know there are clinical trials of Promacta on AA and MDS patients in US, but Hong Kong has not introduced this drug. Our doctor not favour 2nd ATG because of unsatisfactory response to 1st ATG.

We are terrified by the 15% to 20% death rate (quoted by our doctor) for MUD transplant in the first year and the GVHD which could be very serious and complicated. My niece and her mother are too scared to think of the tough road ahead. We are lost and it is difficult to find any AA patients who have gone through BMT or their carers to talk to, as there are very few AA cases in Hong Kong.

Thanks for reading my story, and thanks in advance for any information, advice or sharing, in particular considerations on whether and when to go for BMT, and what we should pay attention to in preparing for transplant.