###Q: wanna see whether this thing is any better at tracking change in the genetic FTD ####LME w/ stnd betas. Compare 2 models. 1 w/ this vari, another with Sbs. Maybe something else. could also calculate change scores. Could do first and last difference, just to see over length of clincal trial which has greater effect size of change. then move to sample size.

###Q: any difference in the longitudinal slope between carriers and non-carrier ####LME. Factor vari with carrier vs non carrier. timeXgroup interaction. W/ plot

###Q: so interested in how this sample size maps onto the sample sizes you’ve seen in the DPM paper ####sample size estimates as noted above. Add to what did for DPM.

save below

Comp score appears to be better able to catch change in GRN as compared to FTLD CDR Sbs.

A little messy though, because I think the FTLD CDR Sbs was used in the creation of this score. Though that might be ok.

Sample size estimates

Tables are split by GRN only vs Any mutation. And baseline ftld CDR of 0.5+ or 1+

GRN+ FTLD CDR 0.5+
Variable	N	Mean	SD	Drug_effect	Treated_Mean	Estimated_Sample_Per_Arm	Avg. Time Interval	Min Time Interval	Max Time Interval
ftldcdr_sb_percent	22	9.131	9.044	30% reduction	6.392	171.112	13.172	10	20
ftldcdr_sb	22	0.249	0.256	30% reduction	0.174	184.458	13.172	10	20
comp_score	22	0.449	0.483	30% reduction	0.314	202.173	13.172	10	20

GRN+ FTLD CDR 1+
Variable	N	Mean	SD	Drug_effect	Treated_Mean	Estimated_Sample_Per_Arm	Avg. Time Interval	Min Time Interval	Max Time Interval
comp_score	10	0.794	0.333	30% reduction	0.555	30.666	13.045	11	15.013
ftldcdr_sb	10	0.429	0.194	30% reduction	0.301	35.625	13.045	11	15.013
ftldcdr_sb_percent	10	10.583	5.942	30% reduction	7.408	54.978	13.045	11	15.013

Mut+ FTLD CDR 1+
Variable	N	Mean	SD	Drug_effect	Treated_Mean	Estimated_Sample_Per_Arm	Avg. Time Interval	Min Time Interval	Max Time Interval
comp_score	69	0.402	0.43	30% reduction	0.281	199.877	13.575	9.921	24
ftldcdr_sb	69	0.224	0.27	30% reduction	0.157	254.712	13.575	9.921	24
ftldcdr_sb_percent	69	4.019	5.828	30% reduction	2.813	366.824	13.575	9.921	24

Mut+ FTLD CDR 0.5+
Variable	N	Mean	SD	Drug_effect	Treated_Mean	Estimated_Sample_Per_Arm	Avg. Time Interval	Min Time Interval	Max Time Interval
comp_score	136	0.251	0.394	30% reduction	0.176	429.054	13.644	9.921	24
ftldcdr_sb	136	0.139	0.234	30% reduction	0.097	493.118	13.644	9.921	24
ftldcdr_sb_percent	136	3.894	7.864	30% reduction	2.726	711.463	13.644	9.921	24

Same as above but this uses a comp_score based on only the GRN and C9 sample.

GRN+ FTLD CDR 0.5+
Variable	N	Mean	SD	Drug_effect	Treated_Mean	Estimated_Sample_Per_Arm	Avg. Time Interval	Min Time Interval	Max Time Interval
ftldcdr_sb_percent	22	9.131	9.044	30% reduction	6.392	171.112	13.172	10	20
ftldcdr_sb	22	0.249	0.256	30% reduction	0.174	184.458	13.172	10	20
comp_score	22	0.434	0.473	30% reduction	0.304	207.088	13.172	10	20

GRN+ FTLD CDR 1+
Variable	N	Mean	SD	Drug_effect	Treated_Mean	Estimated_Sample_Per_Arm	Avg. Time Interval	Min Time Interval	Max Time Interval
comp_score	10	0.768	0.311	30% reduction	0.538	28.526	13.045	11	15.013
ftldcdr_sb	10	0.429	0.194	30% reduction	0.301	35.625	13.045	11	15.013
ftldcdr_sb_percent	10	10.583	5.942	30% reduction	7.408	54.978	13.045	11	15.013

Mut+ FTLD CDR 1+
Variable	N	Mean	SD	Drug_effect	Treated_Mean	Estimated_Sample_Per_Arm	Avg. Time Interval	Min Time Interval	Max Time Interval
comp_score	51	0.401	0.429	30% reduction	0.281	200.195	13.551	10	24
ftldcdr_sb	51	0.246	0.279	30% reduction	0.172	224.422	13.551	10	24
ftldcdr_sb_percent	51	4.885	6.075	30% reduction	3.419	269.748	13.551	10	24

Mut+ FTLD CDR 0.5+
Variable	N	Mean	SD	Drug_effect	Treated_Mean	Estimated_Sample_Per_Arm	Avg. Time Interval	Min Time Interval	Max Time Interval
comp_score	98	0.271	0.398	30% reduction	0.19	376.795	13.596	10	24
ftldcdr_sb	98	0.163	0.246	30% reduction	0.114	396.633	13.596	10	24
ftldcdr_sb_percent	98	5.042	8.505	30% reduction	3.53	496.231	13.596	10	24

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2026-04-16