- Potential for reduced or eliminated complications of SCD (severe pain, strokes, acute chest syndrome) and improved quality of life, without ongoing medical therapy.
- Better outcomes with myeloablative conditioning and matched sibling donors in children, and with haploidentical donors, thiotepa, and post-transplant cyclophosphamide in adults.
- Possible reduced health care usage and burdens long-term.
- Some SCD-related complications may improve after transplantation, although some symptoms or findings may not completely resolve:
- Chronic pain (multifactorial including AVN of the hip)
- Organ dysfunction (some may worsen due to transplant toxicities)
- Quality of life
- Potential for improved life-expectancy.
| - Risk of GVHD or graft failure.
- Risk of myeloid malignancy, especially in case of graft failure.
- Potential for decisional regret later in life about lost fertility or other complications of curative therapy.
- For children, the ultimate disease course is not known.
- Increased risk of regimen-related toxicities, especially if myeloablative conditioning was used and especially in adults.
- For myeloablative conditioning, loss of fertility if tissue cryopreservation or gamete banking is not used.
- High up-front costs.
- Up-front mortality rate of 2 to 5%.
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- New disease-modifying medical therapies continue to be developed.
- Improved transplantation and gene therapy protocols continue to be developed and might be available at a later time.
| - Potential for chronic disease complications including severe pain, strokes, acute chest syndrome, avascular osteonecrosis, loss of fertility, and others.
- Potential for reduced life expectancy, especially for severe disease.
- High long-term health costs and burdens of therapy.
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