This article is more than 5 years old. Information may no longer be current.
HSCT outcomes similar in pediatric ALL regardless of donor type
Click Here to Manage Email Alerts
Pediatric patients with high-risk acute lymphoblastic leukemia who underwent hematopoietic stem cell transplantation with matched unrelated donors demonstrated comparable survival outcomes to those who had matched sibling donors, according to study results.
Christina Peters, MD, of St. Anna Children’s Hospital in Vienna, Austria, and colleagues evaluated data from 411 children with high-risk ALL who underwent hematopoietic stem cell transplantation (HSCT) during a first or later remission. All members of the cohort underwent conditioning with total-body irradiation and etoposide.
“Allogeneic HSCT can effectively induce immunologic antileukemic control in children with ALL by means of the graft-versus-leukemia effect,” Peters and colleagues wrote. “Only 20% to 25% of children with indications for allogeneic HSCT have HLA-matched sibling donors, but the availability of volunteer HLA-matched unrelated donors has increased transplantation rates over recent decades.”
Patients received grafts from HLA-genoidentical siblings when they were available (n = 105), or from a 9/10 or 10/10 HLA-matched unrelated donors (n = 306). Most patients with unrelated donors underwent bone marrow transplantations (69%), whereas 29% received peripheral blood transplantations, and 22% received cord blood alone or in combination with bone marrow or peripheral blood.
Patients with unrelated or sibling donors demonstrated similar rates of 4-year EFS (standard deviation [SD] = 0.67 ± 0.03 vs. 0.71 ± 0.05), 4-year OS (SD = 0.73 ± 0.03 vs. 0.79 ± 0.04) and relapse incidence (SD = 0.22 ± 0.02 vs 0.24 ± 0.04). However, non-relapse mortality occurred more frequently in transplantations from unrelated donors (SD = 0.1 ± 0.02 vs. 0.03 ± 0.02; P = .017).
Among patients with unrelated donors, researchers observed no significant differences in OS, EFS or non-relapse mortality between patients who received 9/10- or 10/10-matched grafts, or between those who underwent peripheral blood or bone marrow transplants.
Post-transplantation neutrophil engraftment occurred more quickly among patients with sibling donors (median time to engraftment, 17 days vs. 22 days), and a greater proportion of patients with a sibling donor achieved neutrophil engraftment (76% vs. 44%; P < .001). Transplantation from a sibling donor also was associated with significantly lower risks for severe infections (16% vs. 39%; P ˂ .001), central neurotoxicity (0% vs. 6%; P = .016) and hypoxia (9% vs. 18%; P = .026).
Incidence and severity of acute graft-versus-host disease (GVHD) were comparable between patients with sibling or unmatched donors; however, the 30-day cumulative incidence of extensive chronic GVHD was higher in patients with sibling donors (0.14 vs. 0.06; P = .012).
Absence of chronic GVHD did not affect EFS rates.
“Our data demonstrate excellent EFS and OS, and low incidence of relapse in children with high-risk ALL after treatment with total-body irradiation and etoposide before allogeneic HSCT from HLA-matched siblings or well-matched unrelated donors,” Peters and colleagues concluded. “This large, prospective, multicenter trial suggests that matched unrelated donor-HSCT could be a standard of care for patients with ALL who have a high risk of relapse and who lack matched sibling donors.” – by Cameron Kelsall
Disclosure: The researchers report honoraria, research funding and travel accommodations from, and consulting/advisory and speakers bureau roles with Amgen, EUSA Pharma, Fresenius Biotech, Genzyme, Medac Pharma, Neovii Biotech, Novartis, Pierre Fabre, Pfizer and RIEMSER Pharma.
Click Here to Manage Email Alerts