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Table 6 Summary of patients undergoing desensitization for drug hypersensitivity

From: Immune responses to alglucosidase in infantile Pompe disease: recommendations from an Italian pediatric expert panel

Sex/age

Phenotype/CRIM status

Symptoms

Immune reaction

Premedication

Desensitization: initial concentration

Desensitization: total dose

Desensitization: adverse reactions

M, 11 months [79]

IOPD CP

Urticarial rash

Elevated serum tryptase, activated complement, IgE and eosinophil count normal

Tranexamic acid, deflazacort, cetirizine, ranitidine

0.5 μg/ml

10 mg/kg (only for the first infusion)

minimal skin reactions

M, 4 months [80]

IOPD CP

Anaphylaxis

IgE and eosinophil count normal

Tranexamic acid, deflazacort, cetirizine, ranitidine

15 μg/ml

Full dose 20 mg/kg EOW

NO

F, 4 months [82]

IOPD, CRIM ND

Urticarial rash

intradermal test positive, SPT negative

Diphenhydramine, methylprednisolone

0.1 μg/ml

20 mg/kg EOW

NO

M, 3 years 8 months [69]

Juvenile onset PD CP

Anaphylaxis

IgG 1:51200, negative IgE titers, complement activation, normal serum tryptase

Diphenhydramine, acetaminophen

0.07 μg/ml

10 mg/kg/week

severe reactions (requiring epinephrine)

M, 10 months [69]

IOPD CP

Urticarial rash

Elevated IgE and serum tryptase, negative IgG and no complement activation

No

0.05 μg/ml

10 mg/kg/weekly

minor skin reaction

F, 6 years 9 months [74]

IOPD CP

Urticarial rash

SPT negative, IgG negative

Prednisolone

0.01 μg/ml

10 mg/kg/weekly

Urticarial rash

F, 15 months [75]

IOPD CP

Anaphylaxis

IgG 1:6400, normal IgE, tryptase and complement

No

1:100

0.2 mg/kg. The dose was doubled every week

No

F 7 months [82]

IOPD CP

Urticarial rash, intractable cough and stridor

Intradermal test positive, SPT negative

No

0.05 μg/ml

10 mg/kg/week

Urticarial rash

F 28 y [56]

LOPD

Anaphylaxis

IgE positive, IgG 1600, SPT negative, intradermal testing positive, complement testing negative, elevated tryptase levels

Diphenhydramine and prednisone

0.1 μg/ml

10 mg/kg/week

severe reactions (requiring epinephrine)

F, 46 years [56]

LOPD

Anaphylaxis

SPT and intradermal tests negative, normal IgE, eosinophil counts, complement, tryptase

Antihistamines H1 (cetirizine), antileukotriene (montelukast)

Drop by drop of ERT solution 2 mg/ml with increasing flow + saline solution with continuous flow by another perfusion pocket

Full dose

Minor reaction: anxiety, crying, abdominal pain

  1. IOPD infantile onset Pompe disease, LOPD late onset Pompe disease, CP CRIM positive, CN CRIM negative, ND not determined, SPT skin prick test, EOW every other week