. Author manuscript; available in PMC: 2020 Nov 5.

Published in final edited form as: Pediatr Blood Cancer. 2019 Mar 22;66(6):e27709. doi: 10.1002/pbc.27709

Table 2:

Phase 2 patient demographic data, laboratory values, and BSs

	Subgroup: Age		Subgroup: Disease severity		Total
	8-9 years	10-12 years	Type 1 VWD	Other disorder
Number of patients	12	17	14	15^†	29
Male gender (%)	7 (58%)	7 (41%)	7 (50%)	7 (47%)	14 (48%)
Mean age years (range)	8.42 (8-9)	10.94 (10-12)	10.14 (8-12)	9.67 (8-12)	9.9 (8-12)
Median Self-PBQ BS (range)	7.5 (2-19)	5 (0-20)	5.5 (0-19)	8 (2-20)	6 (0-20)
Mean VWF:Ag, IU/mL (range)	0.59 (0.17-1.3)	0.43 (0.07-0.98)	0.41 (0.25-0.63)	0.58 (0.07-1.3)	0.48 (0.07-1.30)^*
Mean VWF:RCo, IU/mL (range)	0.42 (0.14-1)	0.36 (0.07-1)	0.30 (0.14-0.41)	0.48 (0.07-1)	0.38 (0.07-1.0)^*
Mean FVIII, IU/mL (range)	0.47 (0.11-0.87)	0.50 (0.26-0.89)	0.62 (0.31-0.89)	0.31 (0.11-0.44)	0.49 (0.11-0.89)^*

Seven patients were missing hemostatic laboratory values (including VWF:Ag, VWF:RCo, and factor VIII:C). Study population means for these categories were calculated using a sample size of 22.

^†

Other bleeding disorders included type 2B VWD (n = 2), type 2M VWD (n = 2), type 2A VWD (n = 1), hemophilia A (n = 4), platelet dysfunction disorders (n = 2), hypofibrinogenemia (n = 1), factor XIII deficiency (n = 1), storage pool deficiencies (n = 1), and Glanzmann’s thrombasthenia (n = 1).