Rapid home therapy infusion of velaglucerase alfa in naïve patients with Gaucher disease

Abstract

AbstractBackgroundEnzyme replacement therapy (ERT) has revolutionised the management of patients with Gaucher disease (GD). In 2018, we published the safety and efficacy of rapid 10‐min infusion of velaglucerase alfa in previously treated patients, mostly on low‐dose therapy.AimTo improve quality of life (QoL) for patients needing lifelong bi‐weekly infusions by introducing a 10‐min infusion instead of 1 h per label in patients naive to ERT and on high‐dose therapy.MethodsFifteen naive patients were enrolled; all received bi‐weekly infusions of 60 units/kgBW velaglucerase alfa; the infusion rate was gradually reduced in the hospital, followed by home infusions. Each infusion was followed for safety. Efficacy parameters were assessed every 3 months. Patient‐reported outcome questionnaires were collected at baseline and follow‐up.ResultsTen‐minute rapid infusions were well tolerated without related severe adverse events (SAEs). Two patients experienced a non‐related SAE and another a possibly related AE. In three patients, the infusion rate was increased to 30 or 60 min (two because of suboptimal response and one because of AE). Two patients dropped out because of an unwillingness to attend follow‐up visits during the COVID‐19 pandemic. All 13 remaining patients reached the 24‐month end‐point. The platelet counts increased by a median (range) of 68.38% (12.5–300%) and the lyso‐Gb1 levels decreased by 62.6% (32.9–89.9%).ConclusionHome therapy with rapid infusion of high‐dose velaglucerase alfa was a safe, effective and preferable alternative for patients with GD naïve to treatment. We believe that shortening the infusion time improves the QoL of patients with GD who have a lifelong commitment to intravenous therapy.