Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation With Post-transplant Cyclophosphamide for Rescuing Patients With Graft Failure

Launched by ASSISTANCE PUBLIQUE - HÔPITAUX DE PARIS · Nov 8, 2021

Keywords

ClinConnect Summary

This clinical trial is looking at a new treatment option for patients who have experienced graft failure after a stem cell transplant. Graft failure means that the transplanted stem cells did not work properly, which can be very serious. The study will test a method called haploidentical stem cell transplantation, where the donor is a family member who shares some but not all of the same genes, along with a medication called post-transplant cyclophosphamide that helps prevent complications. The researchers hope that this approach can improve survival rates for patients facing this challenging situation.

To be eligible for the trial, participants need to be between 3 and 70 years old and must have a hematologic disease, meaning a condition that affects blood or bone marrow. They should have experienced either primary graft failure (failure right after the first transplant) or secondary graft failure (failure within 60 days of the transplant). Participants will also need to have a family member who can serve as a donor and meet certain health criteria. If enrolled, patients can expect close monitoring and care as they receive this new treatment, which the researchers believe could significantly improve their chances of survival compared to existing options.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Aged from 3 to 70 years
• • All hematological diseases
• • Suffering from primary or secondary (within the 60 days post-transplantation) graft failure after a 1st allo-SCT
• * With usual criteria for allo-SCT:
• • ECOG ≤ 2
• • No severe and uncontrolled infection
• • Cardiac function compatible with high dose of cyclophosphamide
• • Adequate organ function: ASAT and ALAT ≤ 2.5N, total bilirubin ≤ 2N, creatinine clearance ≥30ml / min
• • With identification of a haploidentical donor (brother, sister, parents, adult children or cousin)
• • Absence of donor specific antibody (DSA) detected in the patient with a MFI ≥ 1500 (antibodies directed towards the distinct haplotype between donor and recipient)
• • With health insurance coverage (bénéficiaire ou ayant droit).
• • Understand informed consent or optimal treatment and follow-up.
• • Contraception methods must be prescribed during all the duration of the research. Women and men of childbearing age must use contraceptive methods within 12 months and 6 months after the last dose of cyclophosphamide, respectively.
• • Having signed a written informed consent (2 parents for patients aged less than 18)
• Exclusion Criteria:
• • Aged\< 3 years old and \>70 years old
• • With uncontrolled infection
• • With Seropositivity for HIV or HTLV-1 or active hepatitis B or C defined by a positive PCR HBV or HCV and associated hepatic cytolysis
• • Yellow fever vaccine within 2 months before transplantation
• • Cancer in the last 5 years (except basal cell carcinoma of the skin or "in situ" carcinoma of the cervix)
• • Uncontrolled coronary insufficiency, recent myocardial infarction \<6 month, current manifestations of heart failure, uncontrolled cardiac rhythm disorders, ventricular ejection fraction \<50%
• • Heart failure according to NYHA (II or more)
• • Preexisting acute hemorrhagic cystitis
• • Renal failure with creatinine clearance \< 30ml / min
• • Urinary tract obstruction
• • Pregnant (β-HCG positive) or breast-feeding
• • Who have any debilitating medical or psychiatric illness, which preclude understanding the inform consent as well as optimal treatment and follow-up
• • COVID vaccination or recent COVID disease \<3 months
• • Tutorship or curatorship
• • Contraindications to treatments used during the research