Pre-Transplant Immune Suppression With Hematopoietic Cell Transplantation From Haploidentical Donors for Adults and Children With Sickle Cell Disease or ß-Thalassemia (Haplo PTCy)

Summary

Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers think giving participants treatment with fludarabine and dexamethasone, drugs that lower the activity of the body's immune system (immune suppression), before standard conditioning therapy and HCT may help prevent serious side effects, including graft failure and GvHD. In this study, depending on how participants' body responds to the fludarabine and dexamethasone, the study doctor may decide participants should receive another drug, called cyclophosphamide, instead of fludarabine. In addition, depending on the results of participants' routine blood tests, participants may receive the drugs bortezomib and rituximab, which also help with immune suppression.

Eligibility

Age range

2–50 years

Sex

All

Healthy volunteers

No

Interventions

• Drug
  Fludarabine

  PK-guided fludarabine dosing will be used for each of the 2 cycles, using the InsightRx DoseMeRx platform.

• Drug
  Cyclophosphamide

  Cyclophosphamide will be administered Post-Transplant

• Drug
  Tacrolimus

  Tacrolimus will be administered beginning on day +5

• Drug
  Mycophenolate Mofetil

  Mycophenolate mofetil (MMF) will be administered three times daily starting on day +5.

• Biological
  Rabbit ATG

  The dose and schedule of ATG will be determined according to the nomogram in Appendix A

• Drug
  Dexamethasone

  Standard Regimen: Dexamethasone on days -68 to -64 and days -40 to -36.

• Drug

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