for 59 patients, including 26 with MDS. SF3B1 mutation and elevated pre-treatment
expression of aberrant transcripts of Transmembrane Protein 14C (TMEM14C), an SF3B1
splicing target, were observed in MDS subjects who became transfusion free for >8 weeks. Of
the 8 MDS subjects with a TMEM14C AJ/CJ ratio at study entry >4, 7 experienced events of
RBC TI on study (88%). RBC transfusion dependency at study entry by IWG criteria was
confirmed in 5 subjects and SF3B1 mutations were detected in all but one of the 8.
Downregulation of the TMEM14C AJ/CJ ratio with H3B-8800 dosing was also observed with a
nadir at 2-10 hours
Conclusions: H3B-8800 presented an acceptable safety profile and an encouraging rate of
transfusion independence events was observed in a biomarker-defined subset of MDS treated
with this agent.
SCIENTIFIC PROGRAMME
SESSION I
ETIOLOGY OF MDS
SESSION II
BIOLOGY OF MDS –
GENETIC ABNORMALITIES
SESSION III
BIOLOGY OF MDS:
STEM CELLS AND THE
MICROENVIRONMENT
SESSION IV
DIAGNOSTIC WORKUP
AND PROGNOSTIC
FACTORS IN MDS
SESSION V
SPECIFIC SUBTYPES
OF MDS, BASED ON
MORPHOLOGY AND
MOLECULAR BIOLOGY
SESSION VI
TREATMENT OF MDS
SESSION VII
CURRENT PROGRESS IN
THE TREATMENT OF MDS
SESSION VIII
FUTURE TREATMENTS
AND TREATMENT
STRATEGIES IN MDS
SESSION IX
LATE-BREAKING TALKS
SELECTED ABSTRACTS
FOR AN ORAL
PRESENTATION
SELECTED ABSTRACTS
AS E-POSTERS
DISCLOSURES