Table 3 –

GSD 1b Patients and Myelodysplasia or Acute Myeloid Leukemia

Patient ID Number	Year Reported	GSD Mutation	Pre G-CSF ANC Median (Range)	Age at Start of G-CSF Treatment	Years of Treatment with G-CSF	G-CSF Median Dose mcg/kg/day (Range)	Post ANC Median (Range)	Age at MDS/AML
1	2009	Unknown	0.81 (0.27–6.83)	3.8	13.8	0.94 (0.94–1.32)	1.63 (0.01–15.8)	16.7
2	2012	Unknown	0.50 (0.11–0.74)	1.9	18.7	3.18 (1.42–7.18)	1.9 (0.59–12.1)	19.7
3	2017	Unknown	0.44 (0.25–0.85)	3.9	25	6.5 (3.21–6.5)	1.17 (0–3.13)	28.8
4	2012	1211 del CT, IVS8+1 G>A	Unknown	0.42	9.5	(2.3–4.05)	Unknown	10
5	1984	Not tested	Unknown	No G-CSF	No G-CSF	No G-CSF	No G-CSF	4
6	2002	Unknown	0.4 (0.21–0.71)	8	6	2.5 (1.7–4.6)	0.85 (0.7–0.9)	14
7	2008	1211 del CT	Unknown	14	14	2.83	Unknown	28
8	2018	Unknown	Unknown	Infancy	19	Unknown	Unknown	19

Data sources:

#1, 2, 3 Cases from SCNIR-Seattle

#3: Khalaf, et al, Submitted

#4: Case from SCNIR Hannover

#5: Simmons, et al, J Pediatr. 1984;105(3):428–31

#6: Pinsk, et al, J Pediatr Hematol Oncol. 2002;24(9):756–8

#7: Schroeder, et al, J Med Case Rep. 2008;2:319

#8: Li, et al, J Pediatr Hematol Oncol. 2018;35: 45–51