Cessation of enzyme replacement therapy in Gaucher disease

Grinzaid, Karen A.; Geller, Eleanor; Hanna, Soheil L.; Elsas, Louis J.

doi:10.1097/00125817-200211000-00005

Cited by 35 publications

(24 citation statements)

References 15 publications

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“…Thus, in this study, the possible role of genotype, age and splenectomy on the laboratory parameters after ERT reduction was investigated. Among the four patients described by Grinzaid et al, a better clinical and laboratory behaviour was seen in the one with a N370S/N370S genotype after a therapy discontinuation of !1 year (Grinzaid 2002). On the contrary, among the Italian patients, no differences were found in laboratory values variations according to genotype.…”

Section: Discussionmentioning

confidence: 66%

“…However, several authors reported the deterioration of haematological parameters (Hb, Plt, Ct) and an increase in organomegaly (Zimran et al 2011;Grinzaid et al 2002;Vom Dahl et al 2001) after an ERT withdrawal lasting from 3 to more than 24 months. Moreover, Giraldo et al reported conflicting results on 23 patients who withdrew ERT for 6 months, describing stable Hb and Plt in the great number of patients but at the same time the occurrence of a bone crisis in one patient and of mild anaemia in another one (Giraldo et al 2011).…”

Section: Discussionmentioning

confidence: 99%

“…Moreover, although no trials were performed to ascertain the possible effects of a temporary dosage reduction or withdrawal, this issue had already been dealt with before the occurrence of imiglucerase shortage (Drelichman et al 2007;Elstein et al 2000;Grinzaid et al 2002;Schwartz et al 2001;Vom Dahl et al 2001). However, each study analysed only a few patients and the results among the studies were not homogeneous.…”

Section: Introductionmentioning

confidence: 99%

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Did the Temporary Shortage in Supply of Imiglucerase Have Clinical Consequences? Retrospective Observational Study on 34 Italian Gaucher Type I Patients

et al. 2012

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Background. Enzyme Replacement Therapy (ERT) is the standard of care in Gaucher disease. The effects of withdrawal or reduced doses are debated, thus a retrospective cohort study was conducted to investigate clinical and laboratory differences in 34 Gaucher type 1 patients experiencing an ERT dosage reduction after the forced temporary imiglucerase shortage in 2009.Methods. Haemoglobin concentration, leukocytes and platelets counts, and chitotriosidase activity were assessed at baseline and after 6 and 12 months (t0, t6, t12), while bone pain, energy, work or school performance, concentration, memory and social life every 3 months.

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Section: Discussionmentioning

confidence: 66%

Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Did the Temporary Shortage in Supply of Imiglucerase Have Clinical Consequences? Retrospective Observational Study on 34 Italian Gaucher Type I Patients

et al. 2012

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“…92,93 The first and possibly overriding concern was the cost, but beyond this was the question of whether higher doses actually translate into more effective therapy. 94 Moreover, as the above concerns about the inimical aspects of ERT and particularly high-dose ERT are raised, and the question of indications for therapy re-evaluated, the concept of maintenance regimens and/or (intermittent or complete) withdrawal 95,96 and not just dose adjustments 97 after achieving near-normalization of hematological and visceral parameters with ERT should also be re-considered.…”

Section: Unresolved Issuesmentioning

confidence: 99%

Review of the safety and efficacy of imiglucerase treatment of Gaucher disease

Elstein¹

2009

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Abstract:Most patients who suffer from symptomatic Gaucher disease will benefit from enzyme replacement therapy (ERT) with imiglucerase. The safety profile is excellent, only a small percentage of those exposed developing antibodies; similarly, very few patients require pre-medication for allergic reactions. Within 3 to 5 years of imiglucerase therapy, best documented at doses of 30 to 60 units/kg/infusion, hepatosplenomegaly can be expected to be reduced so that the liver volume will be maintained at 1 to 1.5 times normal (30% to 40% reduction from advent of ERT) and spleen volume to 2 to 8 times normal (50% to 60% reduction from advent of ERT). For anemic and thrombocytopenic patients, with 2 to 5 years of imiglucerase, hemoglobin levels are expected to be 11 g/dL for women and children and 12 g/dL for men; and platelet counts in patients with an intact spleen, depending on the baseline value, should approximately be doubled. Bone crises and bone pain but not irreversible skeletal damage will improve in most patients. Nonetheless, some features and some symptomatic patients apparently do not respond equally well and/or perhaps inadequately. The benefit for patients with the neuronopathic forms is primarily in improved visceral and hematological signs and symptoms. There are still several unresolved issues, the high per-unit cost being an important one, which have spurred the development of biosimilar enzymes as well as chaperone therapies currently in clinical trials.

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“…All showed deterioration in hematologic and visceral parameters, and in three patients ERT had to be reinstituted. 28 Elstein suggested that adult patients with stable disease could be withdrawn from ERT for circumscribed periods. However, of the 15 patients who withdrew from ERT for an average of 26 months (range 8-47), six had to restart therapy because of deterioration of clinical features.…”

mentioning

confidence: 99%