Summary of serious infections in patients treated with deferiprone in the postmarketing setting
| Total DFP exposure (111 570.24 patient-y) . | |||||||
|---|---|---|---|---|---|---|---|
| ANC range, ×109/L . | Primary diagnosis . | Infections, n . | Preferred term, Infection term . | Medical history . | Nadir ANC, ×109/L . | Outcome . | Reported cause of death . |
| Group 1 (0.2-0.5) | 5 | ||||||
| β-thalassemia major | Septic shock | Episode of mild leukopenia | 0.4 | Hospitalized, recovered | N/A | ||
| β-thalassemia major | Breast abscess | Severe cardiopathy | 0.4 | Hospitalized, recovered | N/A | ||
| Hereditary hemochromatosis | Fungal sepsis | Inborn hemochromatosis, micronodular cirrhosis, diabetes | 0.2 | Hospitalized, fatal | Septic shock | ||
| β-thalassemia major | Septic shock | No significant history | 0.48 | Hospitalized, fatal | Septic shock | ||
| Diamond Blackfan anemia | Sepsis | Hypothyroidism | 0.27 | Hospitalized, fatal | Sepsis | ||
| Group 2 (0.1-0.199) | 2 | ||||||
| β-thalassemia major | Infectious pleural effusion | No significant history | 0.1 | Hospitalized, recovered | N/A | ||
| β-thalassemia major | Sepsis | Positive serology for Hep C | 0.1 | Hospitalized, fatal | Agranulocytosis and sepsis | ||
| Group 3 (<0.1) | 12 | ||||||
| β-thalassemia major | Oropharyngeal pain | Cardiomyopathy, T1D | 0 | Hospitalized, recovered | N/A | ||
| Aplasia pure red cell | Neutropenic sepsis | Cardiac transplant, end-stage renal failure | 0 | Hospitalized, recovered | N/A | ||
| β-thalassemia major | Mucormycosis | Severe cardiac iron overload, amenorrhea, osteoporosis, extramedullary hematopoiesis | 0 | Hospitalized, recovered with sequelae | N/A | ||
| β-thalassemia major | Herpes simplex | Severe neutropenia on deferiprone therapy | <0.1 | Hospitalized, unknown | N/A | ||
| β-thalassemia major | Septic shock | Hypoparathyroidism | 0 | Hospitalized, fatal | Sepsis | ||
| β-thalassemia major | Rash pustular and septic shock | Regular transfusions | 0 | Hospitalized, fatal | Sepsis | ||
| β-thalassemia major | Sepsis | Hemosiderosis | 0.05 | Hospitalized, fatal | Not reported | ||
| α-thalassemia | Sepsis | Not reported | 0.01 | Hospitalized, fatal | Sepsis | ||
| Secondary hemochromatosis∗ | Furuncle | Lower UTI with right renal lithiasis, paroxysmal cardiac rhythm troubles, dyslipemia | 0.01 | Hospitalized, fatal | Infection | ||
| β-thalassemia major | Sepsis | Failed BMT | <0.1 | Hospitalized, fatal | Sepsis and cardiac arrest | ||
| β-thalassemia major | Sepsis and lymphadenitis | Cardiac hemosiderosis, splenectomy | 0.02 | Hospitalized, fatal | Febrile neutropenia, multiple organ dysfunction syndrome, pneumonia, sepsis | ||
| Congenital sideroblastic anemia | Septic shock and Streptococcal bacteremia | Severe RA, CHF, c.560G > A mutation in SLC25A38 | 0.05 | Hospitalized, fatal | Cardiogenic shock, congestive cardiomyopathy, iron overload, septic shock, agranulocytosis, Streptococcal bacteremia | ||
| Total DFP exposure (111 570.24 patient-y) . | |||||||
|---|---|---|---|---|---|---|---|
| ANC range, ×109/L . | Primary diagnosis . | Infections, n . | Preferred term, Infection term . | Medical history . | Nadir ANC, ×109/L . | Outcome . | Reported cause of death . |
| Group 1 (0.2-0.5) | 5 | ||||||
| β-thalassemia major | Septic shock | Episode of mild leukopenia | 0.4 | Hospitalized, recovered | N/A | ||
| β-thalassemia major | Breast abscess | Severe cardiopathy | 0.4 | Hospitalized, recovered | N/A | ||
| Hereditary hemochromatosis | Fungal sepsis | Inborn hemochromatosis, micronodular cirrhosis, diabetes | 0.2 | Hospitalized, fatal | Septic shock | ||
| β-thalassemia major | Septic shock | No significant history | 0.48 | Hospitalized, fatal | Septic shock | ||
| Diamond Blackfan anemia | Sepsis | Hypothyroidism | 0.27 | Hospitalized, fatal | Sepsis | ||
| Group 2 (0.1-0.199) | 2 | ||||||
| β-thalassemia major | Infectious pleural effusion | No significant history | 0.1 | Hospitalized, recovered | N/A | ||
| β-thalassemia major | Sepsis | Positive serology for Hep C | 0.1 | Hospitalized, fatal | Agranulocytosis and sepsis | ||
| Group 3 (<0.1) | 12 | ||||||
| β-thalassemia major | Oropharyngeal pain | Cardiomyopathy, T1D | 0 | Hospitalized, recovered | N/A | ||
| Aplasia pure red cell | Neutropenic sepsis | Cardiac transplant, end-stage renal failure | 0 | Hospitalized, recovered | N/A | ||
| β-thalassemia major | Mucormycosis | Severe cardiac iron overload, amenorrhea, osteoporosis, extramedullary hematopoiesis | 0 | Hospitalized, recovered with sequelae | N/A | ||
| β-thalassemia major | Herpes simplex | Severe neutropenia on deferiprone therapy | <0.1 | Hospitalized, unknown | N/A | ||
| β-thalassemia major | Septic shock | Hypoparathyroidism | 0 | Hospitalized, fatal | Sepsis | ||
| β-thalassemia major | Rash pustular and septic shock | Regular transfusions | 0 | Hospitalized, fatal | Sepsis | ||
| β-thalassemia major | Sepsis | Hemosiderosis | 0.05 | Hospitalized, fatal | Not reported | ||
| α-thalassemia | Sepsis | Not reported | 0.01 | Hospitalized, fatal | Sepsis | ||
| Secondary hemochromatosis∗ | Furuncle | Lower UTI with right renal lithiasis, paroxysmal cardiac rhythm troubles, dyslipemia | 0.01 | Hospitalized, fatal | Infection | ||
| β-thalassemia major | Sepsis | Failed BMT | <0.1 | Hospitalized, fatal | Sepsis and cardiac arrest | ||
| β-thalassemia major | Sepsis and lymphadenitis | Cardiac hemosiderosis, splenectomy | 0.02 | Hospitalized, fatal | Febrile neutropenia, multiple organ dysfunction syndrome, pneumonia, sepsis | ||
| Congenital sideroblastic anemia | Septic shock and Streptococcal bacteremia | Severe RA, CHF, c.560G > A mutation in SLC25A38 | 0.05 | Hospitalized, fatal | Cardiogenic shock, congestive cardiomyopathy, iron overload, septic shock, agranulocytosis, Streptococcal bacteremia | ||
BMT, bone marrow transplant; CHF, congestive heart failure; Hep C, hepatitis C virus; T1D, type 1 diabetes; N/A, not applicable; RA, rheumatoid arthritis; UTI, urinary tract infection.
The patient was diagnosed with secondary hemochromatosis about a year before the event of agranulocytosis at age 71 years; however, the patient narrative does not include the underlying cause of hemochromatosis.