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Table 3 Summary of adverse eventsa

From: Safety analysis of self-administered enzyme replacement therapy using data from the Fabry Outcome and Gaucher Outcome Surveys

 

Agalsidase alfa

Velaglucerase alfa

 

Self-administered infusion (n = 202)

HCP-supported infusion (n = 2435)b

Self-administered infusion (n = 30)

HCP-supported infusion (n = 814)b

Any AE, n (%)

57 (28.2)

1389 (57.0)

12 (40.0)

271 (33.3)

AE rates per 100PY

7.99

6.78

4.97

4.67

Drug-related AEs, n (%)

12 (5.9)

395 (16.2)

1 (3.3)

19 (2.3)

Serious AEs, n (%)

36 (17.8)

876 (36.0)

6 (20.0)

160 (19.7)

 Blood and lymphatic system disorders

–

18 (0.7)

–

7 (0.9)

 Cardiac disorders

12 (5.9)

315 (12.9)

1 (3.3)

18 (2.2)

 Congenital, familial, and genetic disorders

–

21 (0.9)

–

1 (0.1)

 Ear and labyrinth disorders

–

29 (1.2)

1 (3.3)

1 (0.1)

 Endocrine disorders

–

6 (0.2)

–

1 (0.1)

 Eye disorders

–

13 (0.5)

–

3 (0.4)

 Gastrointestinal disorders

7 (3.5)

124 (5.1)

–

19 (2.3)

 General disorders and administration site conditions

1 (0.5)

139 (5.7)

–

20 (2.5)

 Hepatobiliary disorders

1 (0.5)

23 (0.9)

–

7 (0.9)

 Immune system disorders

–

13 (0.5)

–

–

 Infections and infestations

7 (3.5)

206 (8.5)

2 (6.7)

45 (5.5)

 Injury, poisoning and procedural complications

3 (1.5)

115 (4.7)

1 (3.3)

37 (4.5)

 Investigations

–

39 (1.6)

–

6 (0.7)

 Metabolism/nutrition disorders

–

34 (1.4)

–

2 (0.2)

 Musculoskeletal and connective tissue disorders

2 (1.0)

66 (2.7)

1 (3.3)

24 (2.9)

 Neoplasms

–

62 (2.5)

1 (3.3)

24 (2.9)

 Nervous system disorders

2 (1.0)

254 (10.4)

1 (3.3)

26 (3.2)

 Perinatal, puerperium, and pregnancy-related conditions

3 (1.5)

12 (0.5)

1 (3.3)

5 (0.6)

 Product issues

–

12 (0.5)

–

2 (0.2)

 Psychiatric disorders

2 (1.0)

41 (1.7)

–

6 (0.7)

 Renal and urinary disorders

4 (2.0)

125 (5.1)

–

8 (1.0)

 Reproductive/breast disorders

2 (1.0)

21 (0.9)

–

8 (1.0)

 Respiratory, thoracic, and mediastinal disorders

4 (2.0)

87 (3.6)

–

24 (2.9)

 Skin and subcutaneous tissue disorders

1 (0.5)

18 (0.7)

–

4 (0.5)

 Social circumstances

–

3 (0.1)

–

–

 Surgical/medical procedures

4 (2.0)

16 (0.7)

–

–

 Vascular disorders

2 (1.0)

54 (2.2)

–

4 (0.5)

 Not coded

–

11 (0.5)

–

2 (0.2)

Drug-related serious AEs, n (%)

2 (1.0)c

66 (2.7)

0

1 (0.1)

 Cardiac disorders

–

14 (0.6)

–

–

 Eye disorders

–

1 (< 0.1)

–

–

 Gastrointestinal disorders

–

10 (0.4)

–

–

 General disorders and administration site conditions

–

17 (0.7)

–

–

 Hepatobiliary disorders

–

1 (< 0.1)

–

–

 Immune system disorders

–

2 (0.1)

–

–

 Infections and infestations

–

3 (0.1)

–

–

 Injury, poisoning and procedural complications

–

8 (0.3)

–

–

 Investigations

–

3 (0.1)

–

–

 Musculoskeletal and connective tissue disorders

–

6 (0.2)

–

1 (0.1)

 Neoplasms

–

1 (< 0.1)

–

–

 Nervous system disorders

–

18 (0.7)

–

–

 Respiratory, thoracic, and mediastinal disorders

–

7 (0.3)

–

–

 Skin and subcutaneous tissue disorders

–

3 (0.1)

–

–

 Vascular disorders

–

5 (0.2)

–

–

IRRs, n (%)

9 (4.5)

332 (13.6)

0

13 (1.6)

 Cardiac disorders

1 (0.5)

17 (0.7)

–

–

 Ear and labyrinth disorders

–

6 (0.2)

–

–

 Eye disorders

–

6 (0.2)

–

–

 Gastrointestinal disorders

3 (1.5)

55 (2.3)

–

2 (0.2)

 General disorders and administration site conditions

1 (0.5)

133 (5.5)

–

4 (0.5)

 Hepatobiliary disorders

–

4 (0.2)

–

–

 Immune system disorders

–

14 (0.6)

–

–

 Infections and infestations

–

3 (0.1)

–

–

 Injury, poisoning, and procedural complications

2 (1.0)

69 (2.8)

–

6 (0.7)

 Investigations

–

37 (1.5)

–

–

 Metabolism/nutrition disorders

–

3 (0.1)

–

–

 Musculoskeletal and connective tissue disorders

1 (0.5)

30 (1.2)

–

3 (0.4)

 Neoplasms

–

1 (< 0.1)

–

–

 Nervous system disorders

 

59 (2.4)

–

1 (0.1)

 Psychiatric disorders

–

3 (0.1)

–

–

 Renal and urinary disorders

–

11 (0.5)

–

–

 Respiratory, thoracic, and mediastinal disorders

1 (0.5)

37 (1.5)

–

2 (0.2)

 Skin and subcutaneous tissue disorders

1 (0.5)

50 (2.1)

–

–

 Vascular disorders

1 (0.5)

38 (1.6)

–

–

 Not coded

–

3 (0.1)

–

–

Fatal AEs, n (%)

0

193 (7.9)d

1 (3.3)e

31 (3.8)f

Mean time from start of drug to first treatment-emergent eventg, years

    

 Non-serious AE

2.6

2.9

4.6

4.9

 Serious AE

3.0

4.2

4.5

4.7

 IRR

2.9

2.1

–

2.6

  1. AE, adverse event; ERT, enzyme replacement therapy; HCP, health care provider; IRR, infusion-related reaction; PY, patient years
  2. aAEs were classified according to terminology used in the MedDRA dictionary version 23 and listed by system organ class
  3. bThe HCP-supported populations included all patients who received ≥1 ERT infusion in a clinic/hospital setting or HCP-supported infusion at home, including those patients who later transitioned to self-administration and self-administered ≥1 ERT infusion
  4. cOne patient had two reported drug-related serious AEs of paranoia and abnormal behavior, and another patient had one reported drug-related serious AE of myocardial infarction with agalsidase alfa
  5. dOf these, 5 patient deaths were considered as possibly related to agalsidase alfa (septic shock, lung edema and heart failure, cardiac failure acute/pneumonia, sudden death, and arrhythmia)
  6. eOne patient (age 64) died due to cardiac arrest after 8.2 years of self-administering infusions. This death was not considered to be related to velaglucerase alfa
  7. fNo patient deaths were considered related to velaglucerase alfa
  8. gRefers to mean time from start of drug by each administration methods (self-administered vs HCP-supported)
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172