$ATNM For SIERRA Trial, the primary endpoint is DCR and the secondary endpoint was changed earlier this year to 5-yr overall survival. Those who remember all the FUDing and discussions around Ad Hoc would recall: (a) bashers/bears claiming that no Ad Hoc was exercised as it is a scam and thereby no decisions will be announced (b) bulls were dreaming of DMC recommending Early Halt for futility of conventional care (CC) arm. Bashers/bears fraudulent claim were destroyed. Bulls hopes were also destroyed. SP fell significantly on Dec 28th. The DMC recommended continuation of Trial having examined the unblinded data including dCR. Trial halt historically has been due to futility of control arm, i.e. no significant benefit compared to standard of care or conventional care. Continuation implies dCR could not have been futile. Primary endpoint is 2x dCR for B arm over conventional care arm at 180 days from BMT. Can we estimate this using reliable data? How good are the numbers?.. [1/4]
I share the “Duval Score” – a term used by Gyurcocza et al (Bone Marrow Transplantation volume 52, pages 1083–1090 (2017)] Duval et al p[J. Clin Oncology,2010 ] carried out a retrospective analyses of AML patients who went on to received BMT in 1995-2004. To develop a predictive/prognostic indicator, they considered the following 5 criteria and assigned a value of 0 or 1 to each criterion: (i) Disease group: PIF or duration of first CR; > 6 months (0); <6 months (1) (ii) Cytogenetics prior to HSCT; Good or intermediate (0); Poor (1) (iii) HLA match group (iv) Circulating blasts; absent (0); present (1) (v) Karnofsky or Lansky score; 90-100 (0); <90 (1) The patient pool was then divided in 4 categories based on total score they earned: 0, 1, 2 and 3. Zero being the patient will least complication (if you will). Yellow representing score of zero and red score of 3; I’ve superimposed the CIBMTR data from 2008-2018 as well as SIERRA Trial data. [3/4]
Now, let us look at where we are with respect to other data and predictive models at 100 days mark. I have zoomed in at the top left hand corner of the Figure shared in [1/3] but first the different curves/lines. Is it any good? You decide [4/4]