Evaluation of disease burden and response to treatment in adults with type 1 gaucher disease using a validated disease severity scoring system (DS3)

Table 5 ERT dosing per baseline DS3 severity score category (marked, moderate, mild) when treatment was initiated and at time of last follow up

	ERT dose at initiation of treatment (units/kg/Q2W)			ERT dose at time of last follow-up (units/kg/Q2W)
DS3 category	No. of pts	Median	Range	No. of pts	Median	Range
Marked	58	60.0	8-60	47	50.0	15-120
Moderate	52	52.5	10-60	40	30.0	15-90
Mild	22	60.0	10-60	18	44.0	10-60

All ERT doses are expressed as units/kg body weight infused every 2 weeks (Q2W). For patients who were infused at intervals other than Q2W, the equivalent Q2W dose is shown. ERT dose calculations are inclusive of all ERT agents. At the time of last follow up (or death), 81 % of the initially marked severity patients continued to receive ERT, 77 % of the initially moderate severity patients continued to receive ERT, and 82 % of the initially mild severity patients continued to receive ERT

ISSN: 1750-1172