Table 1.
Treatment Options in Sickle Cell Disease
| Therapy/Agent | Age at Which Approved for Use | Indications | Contra-Indication | Comments |
|---|---|---|---|---|
| Disease Modifying | ||||
| Hydroxyurea | ≥2 years (can start as early as 9 months) | Children, HBSS, HBSβ0 | Pregnancy, breastfeeding | Requires frequent blood tests for monitoring of bone marrow depression, and needs dose reduction in patients with compromised renal function |
| Voxelotor | ≥12 years | for increase of hemoglobin by 1% in any sickle cell disease | End stage renal disease Pregnancy and breast feeding |
Only symptomatic treatment, no change in end organ damage Cost can be prohibitive for use |
| Crizanlizumab | ≥16 years | Reduction of pain crises (if frequency ≥2 per year) | Pregnancy and breast feeding | Requires intravenous administration Only symptomatic treatment, no change in end organ damage Cost can be prohibitive for use |
| L-Glutamine | ≥5 years | To reduce the severity and frequency of complications. | Pregnancy and breast feeding | No change in end-organ damage |
| Chronic Blood Transfusion | Any age | In children with abnormal transcranial doppler velocities, or history of stroke or evidence of cerebrovascular disease on magnetic resonance angiogram. | None | Concerns for iron overload, allo-immunization, hyperviscosity. |
| Curative | ||||
| Hematopoietic Stem Cell Transplant | No specific age, but earlier age improves outcomes. | Stroke, recurrent VOC, Acute chest requiring exchange transfusion, osteonecrosis of multiple joints, sickle nephropathy, red cell alloimmunization (≥2 antibodies) on long term transfusion therapy | Nil | Availability of HLA-matched donor is a serious consideration, treatment related morbidities include including Graft versus host disease, infertility and risk of subsequent malignancy. |