Combination of a haploidentical SCT with an unrelated cord blood unit: a single-arm prospective study

Abstract

We conducted a single-arm prospective study in 50 patients who received the combination of an haploidentical stem cell graft and an unrelated umbilical cord blood unit for the treatment of hematological malignancies. The median time for neutrophil engraftment was 13 days (11-20 days), and for platelets was 15 days (11-180 days). All surviving patients attained complete haploidentical engraftment except three patients who presented a mixed engraftment with increasing cord blood and decreasing haplo mismatch chimerism during the first 4 months after transplantation. The cumulative incidence of grade II-IV acute GVHD was 20% ± 0.327% at day þ 100, and the incidence of chronic GVHD was 19.26% ± 1.0% at 1 year. The 1-year cumulative incidence of relapse was 19.78%±1%, and the TRM was 16.2%±0.54%. At 1 year, overall survival was 78.6%±7.6% and PFS 64.0%±11.0%. The BU/CY-based conditional regimen showed a significant superiority over TBI/CY on PFS (relative risk ¼ 5.012, 95% confidence interval, 1.146-21.927, P ¼ 0.032). In conclusion, the co-infusion of an unrelated cord blood unit may potentially improve the outcome of haploidentical allogeneic hematopoietic SCT.

Combination of a Haploidentical Stem Cell Transplant With Umbilical Cord Blood for Cerebral X-Linked Adrenoleukodystrophy

“…13 Recent data have shown that haploidentical stem cell transplants with support from cord blood, which contains a subset of regulatory T-cell and mesenchymal stromal cells, could significantly reduce the incidences of severe acute GVHD in the treatment of hematological malignancies. 6,14 In this boy, we used haploidentical transplantation with a coinfusion of cord blood to treat CCALD. After transplantation, there was rapid and sustained neutrophil and platelet engraftment, without severe acute GVHD; he showed neurological stability for 6 months.…”

“…5 To avoid this, several studies have adopted haploidentical stem cell transplants with umbilical cord blood infusion to enhance immune reconstitution and prevent GVHD for hematological malignancies. 6 However, very little is known about its use for CCALD patients. This is the first report of a CCALD patient who successfully received a haploidentical stem cell transplant combined with an unrelated umbilical cord blood unit.…”

Alternative Donor Allogeneic Hematopoietic Cell Transplantation for Acute Myeloid Leukemia

“…58 An alternative strategy has involved infusing both an UCB unit and a haplo allograft. 59–61 Although this approach resulted in rapid engraftment, two of the three published studies still showed high NRM of 28% and 35%. Overall, low chronic GVHD and potentially lower relapse with UCBT have made it a promising transplantation platform to continue to refine.…”

Improving The Efficacy And Availability Of Stem Cell Transplant Therapies For Hematopoietic Stem Cell Transplantation

“…This approach has also not reliably demonstrated a reduction in time to donor engraftment, with reported median neutrophil and platelet engraftment times ranging widely between 12-32 days and 41-105 days, respectively [41]. Current clinical trials are exploring new strategies such as co-infusion of haplo-identical CD34 stem cells in the setting of a conventional single or double cord blood unit transplantation (DCBT) [42] to achieve initial rapid neutrophil engraftment from the haploidentical cells, which eventually get rejected while the CB graft gradually takes over hematopoiesis.…”