Table 4.

Summary of patient results

Patient	Sex, age at consent (years)	Splenectomy?	Genotype	Baseline spine DXA Z score	End of study spine DXA Z score	Baseline femur DXA Z score	End of study femur DXA Z score	Last DXA time point (months)	Skeletal eventsa
									Baseline	After
5001	F, 40	N	N370S/N370S	−0.09	−0.10	−1.62	−1.10	12	L
5002	F, 51	Y	?/?	−0.42	−0.40	−0.96	−0.50	48	B	B
5601	M, 32	N	N370S/N370S	−1.29	−1.50	−1.50	−0.80	48
5602	M, 22	N	N370S/N370S	−0.60	0.00	0.72	0.90	48	L	OBLM
5603	M, 53	N	N370S/N370S	3.30b	2.40	1.43	1.20	48
5604	M, 59	N	N370S/N370S	−1.81	−0.90	−1.75	−1.40	48	OB	S
5605	M, 36	N	N370S/N370S	−0.66	0.00	−0.76	0.00	48
5606	M, 22	N	N370S/ISV2+1	0.31	3.90	0.61	−0.30	48	BLM	OLMS
5607	F, 12	N	N370S/N370S	−1.00	−0.20			48	BLM
5608	M, 37	Y	N370S/N370S	−0.36	0.30	−0.88	−0.10	48
5609	M, 42	N	N370S/N370S	−3.20	−3.30	−1.30	−1.20	24	BLM	OM
5701c,d	F, 51	N	N370S/N370S	−2.06	−1.30	−2.19	−1.40	48	LM
5702	M, 38	N	N370S/L444P	−1.47	−0.80	0.31	0.60	48	LM
5703	F, 28	N	N370S/D409H	−0.90	−0.50	−1.80	−1.70	48
5704c	M, 55	N	N370S/N370S	−2.50	−1.20	−0.80	−0.30	36	LM	L
5801	F, 40	N	N370S/?	−0.35	0.05	−1.10	−1.22	12	BLM	BO
6001	M, 68	N	N370S/?	1.30	1.90	3.80	2.90	48	OBLM	LM
6002	M, 64	N	N370S/N370S	−0.04	0.00	0.02	−0.20	36
6201d	F, 44	Y	N370S/L444P	−2.05	−2.20	−1.75	−1.70	48	B
6202	F, 54	N	N370S/N370S	0.76	0.90	−0.49	0.00	48	B
6203d	F, 70	N	N370S/N370S	−0.58	−0.30	−0.68	−0.40	48		L
6204	M, 38	N	N370S/N370S	−1.45		−2.39		BL	B
6205	F, 48	N	N370S/N370S	−0.16	−0.40	−0.54	−0.50	48	M	S
6206	F, 39	Y	N370S/L444P	−2.40	−1.80	−1.70	−1.50	36
6301	M, 41	N	N370S/N370S	−1.39	−0.60	−0.79	−0.20	48
6302	M, 44	N	N370S/N370S	−1.70	−1.60	−1.07	−0.50	48
6303	F, 56	N	N370S/N370S	−0.64		−1.66		BL	LS
6304	M, 64	Y	N370S/N370S	3.38	2.60	0.54	0.50	48
6305	M, 54	N	N370S/L444P	−0.45	−1.40	−0.88	−1.80	48		O
6306d	F, 61	N	N370S/L444P	1.52	1.52	−0.62	−0.47	12	BLM
6307e	M, 34	N	N370S/N370S
6308	F, 38	N	N370S/L444P	−0.96	−0.80	−1.03	−1.00	48	BM
6309	M, 43	N	N370S/N370S	−1.13	−1.10	2.57	2.20	48	BML

DXA, dual-energy X-ray absorptiometry; ?/?, unknown/unknown allele mutations.

^aB, bone crisis; L, lytic lesion; M, medullary infarction; O, osteoarticular necrosis; S, spinal fracture.

^bNo baseline data available. 3.30 is at 24 months.

^cPatient received bisphosphonates (protocol deviation). Patient 5701 received pamidronate from month 21 and patient 5704 received fosamax from month 18.

^dPatient received hormone replacement therapy.

^ePatient 6307 discontinued from the study after one infusion and no baseline scans because of inability to comply with visit schedule.