Clinical data of the 10 patients who required aggressive treatment
| Patients . | Age, y . | Sex . | CD36 status . | Main complications observed during follow-up before the introduction of aggressive treatment . | Treatment . | CSS . |
|---|---|---|---|---|---|---|
| 1 | 10 | F | Negative | Cerebral vasculopathy confirmed by MRA and conventional arteriography | Regular RBC exchange for a year, then BMT | 3 |
| 2 | 8 | M | Negative | Iterative severe painful crises with hospital admission around 34 d/y in the last 2 y | HU | 2 |
| 3 | 12 | M | Negative | Iterative severe painful crises; ACS; avascular necrosis of the head of long bones; sepsis | HU | 8 |
| 4 | 32 | M | Negative | ACS; avascular necrosis of the head of long bones; priapism; papillary necrosis; osteomyelitis; sepsis | Regular RBC exchange | 8 |
| 5 | 25 | M | Positive | ACS (3 episodes); leg ulcerations; papillary necrosis; sepsis | Regular RBC exchange | 11 |
| 6 | 12 | F | Positive | Silent stroke confirmed by MRI; ACS; sepsis | Regular RBC exchange | 7 |
| 7 | 10 | F | Positive | Iterative severe painful crises with hospital admission around 20 d/y in the last 4 y | HU | 2 |
| 8 | 8 | M | Positive | ACS; iterative severe painful crises with hospital admission around 24 d/y in the last 4 y; sepsis | HU | 4 |
| 9 | 11 | M | Positive | Overt stroke and cerebral vasculopathy confirmed by MRA and conventional arteriography; iterative severe painful crisis with hospital admission around 35 d/y in the last 3 y | Regular RBC exchange | 4 |
| 10 | 12 | M | Positive | ACS (4 episodes); sepsis | HU | 8 |
| Patients . | Age, y . | Sex . | CD36 status . | Main complications observed during follow-up before the introduction of aggressive treatment . | Treatment . | CSS . |
|---|---|---|---|---|---|---|
| 1 | 10 | F | Negative | Cerebral vasculopathy confirmed by MRA and conventional arteriography | Regular RBC exchange for a year, then BMT | 3 |
| 2 | 8 | M | Negative | Iterative severe painful crises with hospital admission around 34 d/y in the last 2 y | HU | 2 |
| 3 | 12 | M | Negative | Iterative severe painful crises; ACS; avascular necrosis of the head of long bones; sepsis | HU | 8 |
| 4 | 32 | M | Negative | ACS; avascular necrosis of the head of long bones; priapism; papillary necrosis; osteomyelitis; sepsis | Regular RBC exchange | 8 |
| 5 | 25 | M | Positive | ACS (3 episodes); leg ulcerations; papillary necrosis; sepsis | Regular RBC exchange | 11 |
| 6 | 12 | F | Positive | Silent stroke confirmed by MRI; ACS; sepsis | Regular RBC exchange | 7 |
| 7 | 10 | F | Positive | Iterative severe painful crises with hospital admission around 20 d/y in the last 4 y | HU | 2 |
| 8 | 8 | M | Positive | ACS; iterative severe painful crises with hospital admission around 24 d/y in the last 4 y; sepsis | HU | 4 |
| 9 | 11 | M | Positive | Overt stroke and cerebral vasculopathy confirmed by MRA and conventional arteriography; iterative severe painful crisis with hospital admission around 35 d/y in the last 3 y | Regular RBC exchange | 4 |
| 10 | 12 | M | Positive | ACS (4 episodes); sepsis | HU | 8 |
CSS indicates clinical severity score; MRA, magnetic resonance angiography; RBC, red blood cell; BMT, bone marrow transplantation; HU, hydroxyurea; ACS, acute chest syndrome; MRI, magnetic resonance imaging.