Successful Desensitization to Imiglucerase of an Adult Patient Diagnosed with Type I Gaucher Disease

Erdoğdu, Derya; Gelincik, Aslı; Canbaz, Bülent; Çolakoğlu, Bahattin; Büyüköztürk, Suna; Tanakol, Refik

doi:10.1159/000339754

Cited by 11 publications

(5 citation statements)

References 22 publications

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“…However, when these measures fail to prevent hypersensitivity reactions, desensitisation with the culprit enzyme is indicated [9]. In the literature, a few patients have received ERT with desensitisation [3,5,6,7,10,11,12]. Here we reported three cases in which the patients had IgE-mediated hypersensitivity reactions and subsequently underwent desensitisation prior to each application of ERT, without further reactions.…”

Section: Discussionmentioning

confidence: 99%

IgE-Mediated Hypersensitivity and Desensitisation with Recombinant Enzymes in Pompe Disease and Type I and Type VI Mucopolysaccharidosis

Çapanoğlu¹,

Mısırlıoğlu²,

Azkur³

et al. 2016

Int Arch Allergy Immunol

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Enzyme replacement therapy (ERT) is important for the treatment of lysosomal storage disorders. Hypersensitivity reactions with ERT have been reported, and in these cases, desensitisation with the enzyme is necessary. Here we report the cases of 3 patients with lysosomal storage disorders, including Pompe disease and mucopolysaccharidosis type I and VI, who had IgE-mediated hypersensitivity reactions and positive skin tests. Successful desensitisation protocols with the culprit enzyme solution were used for these patients. All 3 patients were able to safely receive ERT with the desensitisation protocol.

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Section: Discussionmentioning

confidence: 99%

IgE-Mediated Hypersensitivity and Desensitisation with Recombinant Enzymes in Pompe Disease and Type I and Type VI Mucopolysaccharidosis

Çapanoğlu¹,

Mısırlıoğlu²,

Azkur³

et al. 2016

Int Arch Allergy Immunol

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“…A 12-step standard rapid desensitization protocol was used for immediate reactions [2] and prolonged protocols were used for nonimmediate reactions according to the previously described protocols. Tailor made protocols were generated where no related protocols had been published for individual drugs, as shown in Table 1 [13141516171819].…”

Section: Methodsmentioning

confidence: 99%

Interleukin-10 is increased in successful drug desensitization regardless of the hypersensitivity reaction type

Gelincik

Demir

Şen

et al. 2019

Asia Pacific Allergy

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BackgroundLittle is known about the mechanism of desensitization in hypersensitivity drug reactions.ObjectiveThe aim of this study was to evaluate the effects of drug desensitization on some cytokine levels in patients desensitized for drug hypersensitivity reactions.MethodsPatients with a hypersensitivity reaction to any drug for whom desensitization was planned with the culprit drug, patients who could tolerate the same drugs and healthy subjects who were not exposed to these drugs were enrolled. Bead-based Milliplex MAP multiplex technology was used to determine interleukin (IL)-4, IL-5, interferon-γ and IL-10 levels in the sera of the subjects as a baseline and 24 hours after desensitization had been completed in the patients.ResultsA total of 26 patients (16 female [61.5%]; mean age 48.46 ± 15.97 years old), 10 control patients (5 female [50%]; mean age 47.4 ± 15.4 years old) and 5 healthy subjects (3 female [60%]; mean age 34.2 ± 5.6 years old) were enrolled. Four of the 26 patients did not tolerate the procedure and were grouped as the ‘unsuccessful desensitization group’ whereas 22 patients successfully completed the procedure and formed the ‘successful desensitization group.’ Baseline cytokine levels in the 3 groups were not statistically different. Postdesensitization IL-10 levels in the successful desensitization group were significantly higher than their initial levels (p = 0.005) whereas none of the cytokine levels significantly changed in the unsuccessful desensitization group. The rise in IL-10 levels was greater in chemotherapeutic desensitizations when compared to other drugs (p = 0.006).ConclusionSuccessful desensitization independent of the hypersensitivity reaction type seems to be related to the increase of IL-10.

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“…These side effects are usually mild [26, 27], allowing continuation of therapy after slowing of infusion rates or the use of pharmacological support with corticosteroids and antihistamines. In patients who respond poorly to these treatments, a desensitization protocol has successfully been implemented in both pediatric and adult patients [28, 29]. Unfortunately, despite many attempts, premedication with methylprednisolone and chlorpheniramine maleate did not result in improved toleration of imiglucerase infusions in our patient.…”

Section: Discussionmentioning

confidence: 90%

Successful switch from enzyme replacement therapy to miglustat in an adult patient with type 1 Gaucher disease: a case report

et al. 2016

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BackgroundGaucher disease is one of the most common lipid-storage disorders, affecting approximately 1 in 75,000 births. Enzyme replacement therapy with recombinant glucocerebrosidase is currently considered the first-line treatment choice for patients with symptomatic Gaucher disease type 1. Oral substrate reduction therapy is generally considered a second-line treatment option for adult patients with mild to moderate Gaucher disease type 1 who are unable or unwilling to receive lifelong intravenous enzyme infusions. The efficacy and safety of the oral substrate reduction therapy miglustat (Zavesca®) in patients with Gaucher disease type 1 have been established in both short-term clinical trials and long-term, open-label extension studies. Published data indicate that miglustat can be used as maintenance therapy in patients with stable Gaucher disease type 1 switched from previous enzyme replacement therapy.Case presentationWe report a case of a 44-year-old Caucasian man with Gaucher disease type 1 who was initially treated with enzyme replacement therapy but, owing to repeated cutaneous allergic reactions, had to be switched to miglustat after several attempts with enzyme replacement therapy. Despite many attempts, desensitization treatment did not result in improved toleration of imiglucerase infusions, and the patient became unwilling to continue with any intravenous enzyme replacement therapy. He subsequently agreed to switch to oral substrate reduction therapy with miglustat 100 mg twice daily titrated up to 100 mg three times daily over a short period. Long-term miglustat treatment maintained both hemoglobin and platelet levels within acceptable ranges over 8 years. The patient’s spleen volume decreased, his plasma chitotriosidase levels stayed at reduced levels, and his bone mineral density findings have remained stable throughout follow-up. The patient’s quality of life has remained satisfactory. Miglustat showed good gastrointestinal tolerability in this patient, and no adverse events have been reported.ConclusionsOral miglustat therapy proved to be a valid alternative treatment to intravenous enzyme replacement therapy for long-term maintenance in this patient with Gaucher disease type 1, who showed persistent allergic intolerance to imiglucerase infusions. This report exemplifies the type of patient with Gaucher disease type 1 who can benefit from switching from enzyme replacement therapy to substrate reduction therapy.

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Successful Desensitization to Imiglucerase of an Adult Patient Diagnosed with Type I Gaucher Disease

Cited by 11 publications

References 22 publications

IgE-Mediated Hypersensitivity and Desensitisation with Recombinant Enzymes in Pompe Disease and Type I and Type VI Mucopolysaccharidosis

IgE-Mediated Hypersensitivity and Desensitisation with Recombinant Enzymes in Pompe Disease and Type I and Type VI Mucopolysaccharidosis

Interleukin-10 is increased in successful drug desensitization regardless of the hypersensitivity reaction type

Successful switch from enzyme replacement therapy to miglustat in an adult patient with type 1 Gaucher disease: a case report

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