Mode
Text Size
Log in / Sign up

Low-dose ATG plus PTCy shows no significant difference in acute GVHD compared to standard regimensTrial Shows New Treatment Strategy for Leukemia Stem Cell Transplant

AI-generated summary of the cited source, checked by automated accuracy review. How we work

Key Takeaway
Note that low-dose ATG plus PTCy provides similar acute GVHD protection and survival as standard-dose regimens.

This randomized trial evaluated 407 patients aged 14 to 70 years with acute myeloid leukemia or myelodysplastic syndromes undergoing haploidentical peripheral blood stem cell transplantation. The study compared low-dose antithymocyte globulin (ATG; 5 mg/kg) plus posttransplant cyclophosphamide (PTCy; 50 mg/kg) against standard-dose ATG (10 mg/kg) or a PTCy-based regimen (100 mg/kg).

The primary outcome was the cumulative incidence (CI) of grade 2 to 4 acute GVHD by day 100 and GVHD-free, relapse-free survival at 1 year. Results showed that the CI of grade 2 to 4 acute GVHD did not differ significantly among the 3 groups (P =.210). Survival outcomes also showed no significant differences across the three regimens.

Secondary outcomes included chronic GVHD and recovery rates. The overall incidence of chronic GVHD was comparable across all groups (P =.110). While the 2-year CI of moderate--to-severe cGVHD was numerically lower in the ATG/PTCy (17.4%) and ATG (17.3%) groups than the PTCy group (28.3%), this did not reach statistical significance (P =.095). However, the CI of neutrophil and platelet recovery was significantly higher in the ATG/PTCy group compared to other groups (P <.001).

Safety data regarding specific adverse events were not reported. The trial suggests that three GVHD prophylaxis strategies provide similar efficacy in preventing acute GVHD and maintaining survival. Clinicians may consider these regimens as comparable options for managing graft-versus-host disease risk in this patient population.

How this fits prior evidence

How this fits prior evidence: This finding addresses a gap in the management of myelodysplastic syndromes (MDS) by evaluating specific prophylaxis regimens. While previous coverage noted that Luspatercept and ESA-based regimens show higher rates of erythroid haematological improvement in lower-risk MDS, this trial focuses on the efficacy of ATG and PTCy combinations for graft acceptance. The results confirm that multiple GVHD prophylaxis strategies provide comparable outcomes for acute GVHD and survival in AML and MDS patients undergoing haploidentical transplantation.

Researchers conducted a Phase 3 trial involving 407 patients aged 14 to 70. These patients had acute myeloid leukemia or myelodysplastic syndromes and were undergoing haploidentical peripheral blood stem cell transplants. The study compared three different ways to prevent graft-versus-host disease, which is a common complication where the body attacks its own tissues.

The trial found that all three treatment groups performed similarly in preventing severe acute reactions and maintaining survival rates at one year. However, patients who received a specific combination of low-dose antithymocyte globulin and posttransplant cyclophosphamide showed significantly faster recovery of neutrophils and platelets compared to the other two groups.

While some numbers suggested lower rates of chronic issues in certain groups, these results were not statistically significant. Because this study shows that different methods provide similar protection against immediate complications but vary in how quickly they restore blood counts, patients should talk to their doctors about which approach best fits their specific recovery needs.

What this means for you:
A new drug combination may help some transplant patients recover blood cells faster while providing similar safety levels.

Common questions

How does this treatment help with recovery?

The study found that the group receiving low-dose antithymocyte globulin and posttransplant cyclophosphamide had significantly higher rates of neutrophil and platelet recovery compared to the other groups. This means their blood counts may recover more quickly after the transplant.

Is this treatment safer than others?

The trial showed that all three groups had similar rates of severe acute graft-versus-host disease and comparable survival outcomes at one year. While some results for chronic issues were numerically lower in certain groups, they were not statistically different from the other treatments.

Who is eligible for this type of study?

The trial included 407 patients between the ages of 14 and 70. These individuals were specifically diagnosed with acute myeloid leukemia or myelodysplastic syndromes with excess blasts I or II who were undergoing a haploidentical peripheral blood stem cell transplant.

Study Details

Study typeRct
EvidenceLevel 2
Follow-up840.0 mo
PublishedJun 2026
View Original Abstract ↓
The optimal graft-versus-host disease (GVHD) prophylaxis strategy in haploidentical peripheral blood stem cell transplantation remains controversial. In this open-label, phase 3 study, patients aged 14 to 70 years with acute myeloid leukemia or myelodysplastic syndromes with excess blasts Ⅰ or Ⅱ were randomized (2:1:1) to receive low-dose antithymocyte globulin (ATG; 5 mg/kg) plus posttransplant cyclophosphamide (PTCy; 50 mg/kg; referred to as ATG/PTCy), standard-dose ATG (total dose, 10 mg/kg), or a PTCy-based (total dose, 100 mg/kg) regimen for GVHD prophylaxis. The coprimary end points were the cumulative incidence (CI) of grade 2 to 4 acute GVHD (aGVHD) by day 100 and GVHD-free, relapse-free survival at 1 year after transplant. A total of 407 patients were randomized to receive an ATG/PTCy (185 patients), ATG (113 patients), or PTCy (109 patients) regimen for GVHD prophylaxis. By day +100, the CI of grade 2 to 4 aGVHD did not differ significantly among the 3 groups (P = .210). Although the overall incidence of chronic GVHD (cGVHD) was comparable across all groups (P = .110), the 2-year CI of moderate-to-severe cGVHD was numerically lower in the ATG/PTCy (17.4%) and ATG (17.3%) groups than the PTCy group (28.3%), without reaching statistical significance (P = .095). No significant differences were observed in survival outcomes among the 3 groups. Notably, the CI of neutrophil and platelet recovery was significantly higher in the ATG/PTCy group than in the other groups (P< .001). This trial suggested that the 3 GVHD prophylaxis strategies presented similar efficacy in preventing grade 2 to 4 aGVHD and yielded comparable survival. This trial was registered at www.clinicaltrials.gov as NCT03608059.
Free Newsletter

Clinical research that matters. Delivered to your inbox.

Join thousands of clinicians and researchers. No spam, unsubscribe anytime.