Treatment for graft failure after stem cell transplant

Treatment of Graft Failure After Hematopoietic Stem Cell Transplantation

This study is testing a new treatment for patients who have had a stem cell transplant and are experiencing graft failure, to see if a combination of medications and radiation can help the donor cells take hold in their body.

Quick facts

• Study type: Observational
• Enrollment: 50 (estimated)
• Sex: All
• Sponsor: Masonic Cancer Center, University of Minnesota (Academic / other)
• Drugs / interventions: chemotherapy, cyclophosphamide, fludarabine
• Locations: 1 site (Minneapolis, Minnesota)
• Trial ID: NCT02161783 on ClinicalTrials.gov

What this trial studies

This observational study focuses on treating graft failure following hematopoietic stem cell transplantation (HSCT) using a regimen of cyclophosphamide, fludarabine, and low-dose total body irradiation. The goal is to promote donor engraftment by day 42 after initial graft failure. The source of stem cells will be determined by the transplant team based on patient-specific factors such as age and medical history. This approach aims to provide a second chance for patients experiencing primary or secondary graft failure.

Who should consider this trial

Good fit: Ideal candidates include patients with primary or secondary graft failure who meet specific organ function criteria.

Not a fit: Patients who do not have graft failure or those with unacceptable organ function may not benefit from this treatment.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the chances of engraftment and recovery for patients who have experienced graft failure.

How similar studies have performed: Other studies have explored similar treatment regimens for graft failure, showing promising results, but this specific approach may offer novel insights.

Eligibility criteria

Inclusion Criteria:

* Patients with primary or secondary graft failure, as defined below, may receive a second transplant:

  * Primary graft failure is defined as not achieving an ANC ≥0.5x10\^9/L for three consecutive days by day 35 - 42 following the first transplant.
  * Secondary graft failure is defined as achieving an ANC ≥0.5x10\^9/L for three consecutive days by day 35 - 42, but subsequently drops below 0.5x10\^9/L without recovery.
  * Loss of chimerism is defined as achieving an ANC ≥0.5x10\^9/L for three consecutive, but with less than 10% CD15+ donor cells in the marrow or peripheral blood.
* Recipients should have acceptable organ function defined as:

  * Renal: creatinine \< 2.0 (adults) and creatinine clearance \> 30. For creatinine clearance \< 70, consultation with a BMT pharmacist is necessary for chemotherapy dose adjustments.
  * Hepatic: bilirubin, AST/ALT, ALP \< 10 x upper limit of normal
  * Cardiac: left ventricular ejection fraction \> 40%

Exclusion Criteria:

* Uncontrolled infection at the time of transplant.
* Patients with Fanconi Anemia or other DNA breakage syndromes.

Where this trial is running

Minneapolis, Minnesota

• University of Minnesota Medical Center, Fairview — Minneapolis, Minnesota, United States (Recruiting)

Study contacts

• Principal investigator: Troy C Lund, MD, PhD — University of Minnesota
• Study coordinator: Timothy Krepski

Conditions Primary Graft Failure; Secondary Graft Failure; Hematopoietic stem cell transplant