Splenectomy in Gaucher Disease: New Management Dilemmas 722

Krasnewich, Donna M.; Hill, Suvimol; Dietrich, Kathleen; Bauer, Larry; Ginns, Edward I.; Sidransky, Ellen

doi:10.1203/00006450-199804001-00743

Cited by 8 publications

(7 citation statements)

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“…Splenectomy should only be considered in exceptional circumstances after assessment by a physician experienced in the management of Gaucher disease. These circumstances may include, for example, when unexplained masses are present in the spleen (Hughes et al 2007;Krasnewich et al 1998) and/or in cases of refractory severe thrombocytopenia associated with spontaneous bleeding in vital organs.…”

Section: Splenectomymentioning

confidence: 98%

Management of non‐neuronopathic Gaucher disease with special reference to pregnancy, splenectomy, bisphosphonate therapy, use of biomarkers and bone disease monitoring

Cox¹,

Aerts²,

Belmatoug³

et al. 2008

J of Inher Metab Disea

121

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Enzyme replacement was introduced as treatment for non-neuronopathic Gaucher disease more than 15 years ago. To ensure the best use of this costly ultra-orphan agent, a systematic disease management approach has been proposed by an international panel; this includes the development, by consensus, of achievable treatment goals. Here we critically review these goals and monitoring guidelines and incorporate emerging experience of the disease in the therapeutic era, as well as contemporary clinical research. This review makes recommendations related specifically to the management of pregnancy; the appropriate use of splenectomy and bisphosphonate treatment; the relevance of biochemical markers to disease monitoring; and the use of semi-quantitative methods for assessing bone marrow infiltration. In addition, we identify key areas for development, including the requirement for a validated index of disease severity; the need to correlate widely used biomarkers with long-term disease outcomes, and the desirability of establishing agreed standards for monitoring of bone disease particularly in infants and children with Gaucher disease.

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Section: Splenectomymentioning

confidence: 98%

Management of non‐neuronopathic Gaucher disease with special reference to pregnancy, splenectomy, bisphosphonate therapy, use of biomarkers and bone disease monitoring

Cox¹,

Aerts²,

Belmatoug³

et al. 2008

J of Inher Metab Disea

121

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“…Total splenectomy should therefore be avoided as far as possible. However, it may occasionally be necessary in order to improve response to therapy (Krasnewich et al 1998). In this situation we would recommend a partial rather than a total splenectomy (Altarescu et al 2001).…”

Section: Monitoringmentioning

confidence: 98%

Management of neuronopathic Gaucher disease: Revised recommendations

Vellodi

Tylki‐Szymańska

Davies³

et al. 2009

J of Inher Metab Disea

121

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The original guidelines drawn up for the management of the neuronopathic forms of Gaucher disease were felt to be in need of revision; in particular, the role of high-dose enzyme replacement therapy (120 IU/kg of body weight every 2 weeks) in stabilizing neurological disease. The existing published evidence was analysed; it was concluded that it did not support the role of high-dose ERT, although this might be required to treat severe visceral disease.

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“…Elle permet de gérer les complications liées au syndrome tumoral et à son retentissement en terme de douleur et d'envahissement local, aux cytopénies et au syndrome hémorragique. Elle est actuellement réservée aux échecs du TES et aux situations d'urgence [41].…”

Section: Traitement Symptomatiqueunclassified