Febrile neutropenia 83 81 84 83
Petechiae 36 27 1 1
Nervous system disorders
Headache a 46 38 3 3
Musculoskeletal and connective tissue disorders
Musculoskeletal pain a 33 31 5 2
Arthralgia 14 8 < 1 < 1
Respiratory, thoracic and mediastinal disorders
Epistaxis 28 24 3 1
Infections and infestations
Device-related infection 24 17 16 10
Upper respiratory tract infection a 20 15 4 3
Investigations
Hyperglycemia a 20 17 7 6
Activated partial thromboplastin time prolonged 13 8 3 2
Skin and subcutaneous tissue disorders
Hyperhidrosis 14 8 0 0
Renal and urinary disorders
Renal insufficiency a 12 9 5 3
Psychiatric disorders
Insomnia 12 8 0 < 1
1 For trial sites in North America, only Grades 3 and 4 were collected.
a based on grouping of individual PTs:
• Upper respiratory tract infections: e.g. nasopharyngitis, upper respiratory tract infections, sinusitis
• Mucositis: e.g. radiation mucositis, stomatitis, laryngeal pain
• Musculoskeletal pain: e.g. back pain, bone pain, pain in extremity
• Renal insufficiency: e.g. blood creatinine increased, renal failure, acute kidney injury
• Hyperglycemia: mainly hyperglycemia
Other notable adverse reactions occurring in less than 10% of patients treated with RYDAPT but at least 2% more frequently than in the placebo group included:
Infections and infestations: Cellulitis a (7%), fungal infectiona (7%)
Metabolism and nutrition disorders: Hyperuricemia (8%)
Nervous system disorders: Tremor (4%)
Eye disorders: Eyelid edema (3%)
Cardiac disorders: Hypertensiona (8%), pericardial effusion (4%)
Respiratory, thoracic and mediastinal disorders: pleural effusion (6%)
Skin and subcutaneous tissue disorders: Dry skin (7%)
General disorders and administration site conditions: Thrombosisa (5%)
Investigations: Weight increased (7%), hypercalcemia (3%)
a based on grouping of individual PTs:
Thrombosis: e.g. thrombosis in device, thrombosis
Cellulitis: e.g. cellulitis, erysipelas
Fungal infection: e.g. Bronchopulmonary aspergillosis, pneumonia fungal, splenic infection fungal, hepatic candidiasis
Table 3: New or Worsening Laboratory Abnormalities (≥ 10% Incidence and ≥ 2% More Frequent on the Midostaurin Arm) Reported in Patients with AML on Study 1
Laboratory Abnormality RYDAPT
(50 mg twice daily)
N=345
All Grades
% RYDAPT
(50 mg twice daily)
N=345
Grade 3/4
% Placebo
(N=335)
All Grades
% Placebo
(N=335)
Grade 3/4
%
Alanine aminotransferase (ALT) increased&
