Curative Treatments

Review and compare all of the curative treatments for Sickle Cell Disease

Full Match Bone Marrow Transplant

This procedure has been around for longer than all of the other options. With a full sibling match success rate is 95%+.

- First Performed in 1983

- Requires Chemotherapy

- Has the risk of fertility loss

-Temporary hair loss

- Temporary low immune system

- Uses a full sibling match or 10/10 unrelated match from a database of donors.

-No Age Requirement. Requirement is based on severity of condition and health.

- Process takes 3 months for Adults and 6 months for children

- Success rate is 95% with a sibling donor
85% with an unrelated donor

FIND PROVIDERS

Haploid Identical Bone Marrow Transplant

If a full match donor is not available and you are unable to access gene therapy, this is a good option. You can get a half match from your mother, father or sibling.

- First Performed in 2011

- Requires Chemotherapy

- Has the risk of fertility loss

-Temporary hair loss

- Temporary low immune system

- Uses a half or 50% match from a parent or sibling.

-No Age Requirement. Requirement is based on severity of condition and health.

- Process takes 3 months for Adults and 6 months for children

-Success rate is 88%

FIND PROVIDERS

Gene Therapy

Gene Therapy is a great option because you do not have to be reliant on anyone to be a donor. Gene Therapy uses the patient's own cells and modifies them, it also has 95%+ success rate.

- First Performed in 2015

- Requires Chemotherapy

- Has the risk of fertility loss

-Temporary hair loss

- Temporary low immune system

- Uses the patient's own cells and the cells are modified.

-Must be at least 12 years old

- Process takes 8-12 months

-Success rate is 95%+

FIND PROVIDERS

Things to Consider

If you have a full sibling match or a 10/10 unrelated match a Full Match Bone Marrow Transplant may be your best option.
If you do not have a full sibling match or any 10/10 donors available haploid identical transplant or gene therapy should be considered.
Gene therapy has a high success rate, however you must be 12 years of age or older and must be willing for the process to take 8 to 12 months.
Gene therapy for sickle cell has been around since 2015 while its counter parts have been around longer.
Curative treatments are typically performed on children who are 16 and younger who have complications from their sickle cell disease who still have healthy organ function. This is not to say that adults living with sickle cell cannot get a transplant or gene therapy, this is dependent on their health and their medical provider's discretion.
Please consult with multiple medical professionals before making any decisions.

Sources of Information

https://www.hopkinsmedicine.org/news/newsroom/news-releases/2023/12/a-cure-for-sickle-cell-disease

https://www.chop.edu/news/researchers-publish-final-results-key-clinical-trial-gene-therapy-sickle-cell-disease

https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease#:~:text=The%20safety%20and%20effectiveness%20of%20Lyfgenia%20is%20based%20on%20the,CR%20during%20this%20time%20period.

https://www.yalemedicine.org/news/gene-therapies-sickle-cell-disease

https://crisprtx.com/focus-areas/hemoglobinopathies

https://www.healthline.com/health/is-there-a-cure-for-sickle-cell-anemia#issues

https://www.nejm.org/doi/full/10.1056/NEJM199608083350601

https://dhhr.wv.gov/bph/programs/Documents/BMT-for-sickle-cell-disease.pdf

https://www.stjude.org/care-treatment/treatment/blood-disorders/sickle-cell/sickle-cell-disease.html#:~:text=Jude%20patient%20was%20the%20first,children%20with%20sickle%20cell%20disease.