Medal lecture: Judith Marsh on How I treat aplastic anaemia #BSH2017
When assessing new pts don't discount 'asthma' or 'alcoholic liver dx' which may be pulm fibrosis or cirrhosis due to BMF syndrome #BSH2017
Disease severity is clearly outlined in the @BritSocHaem guidelines onlinelibrary.wiley.com/doi/10.1111/j.… #BSH2017
Hypocellular MDS needs to be ruled out - increased myeloid precursors, increased reticulin or abnormal megas are suspicious #BSH2017
Response rate w rabbit ATG inferior - no longer recommended (37% vs 68% at 6/12) #BSH2017
Infection the most common cause of death after ATG. Cardiac events should not be underestimated #BSH2017
PIGA and BCOR mutns infer better outcomes but others such as DNMT3a or ASXL1 do worse #BSH2017
Response to ATG is independent of age in adults #BSH2017
Whether to do transplant up front for severe AA w sib donor should not be limited by age 50 if pt older and considered fit #BSH2017
Data in children with AA shows outcomes from MUD=sib alloSCT #BSH2017
In non severe AA use of ATG w CSA increases speed and duration of response vs CSA alone #BSH2017
Constitutional bone marrow failure syndromes may go unrecognised and present late in life #BSH2017
Important as can be targeted specifically eg danazol for 'telomeropathies' #BSH2017
Must avoid using related and also-affected donors for HSCT #BSH2017
Also must ensure ongoing cancer surveillance as these pts high risk #BSH2017
Response rates for 2nd ATG+CSA at best 30% & increase risk of AML/MDS later on #BSH2017
Eltrombopag is a small molecule and can enter stem cell niche. May also have off target effects eg on Treg cell function #BSH2017
When critical no. HSCs respond after eltrombopag, haemopoiesis may be sustained after stopping the drug #BSH2017
But caution as 19% develop clonal evolution #BSH2017
Is there a role for upfront MUD in adults with sAA and no sib donor? Prof Marsh argues yes - 1y TRM 5% -no higher than ATG outcomes #BSH2017
Recipient HLA abs against donor ags precludes use of that donor -need to consider when giving blood product support pre transplant #BSH2017
ATG, CSA + eltrombopag appears to give lower risk of clonal evolution (8%) #BSH2017
RACE study compares horse ATG, CSA +/- eltrombopag as front line rx for SAA hematologiegroningen.nl/protocollen/do… #BSH2017
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