Studies using PTCy or abatacept for GVHD prophylaxis for MMUD HCT
| Study . | Population . | Design . | Comparison . | Key findings . |
|---|---|---|---|---|
| Shaw et al (2021)35 | 80 patients who underwent RIC/MAC BMSC MMUD HCT with PTCY/MMF/sirolimus-based GVHD prophylaxis | Multicenter, prospective, phase 2 trial | NA | 1. The 1-year OS was 76% (72% for MAC and 79% for RIC) 2. Degree of HLA mismatch did not affect the OS (75% for 7/8 and 77% for 4-6/8). 3. The day +100 incidence of grade II to IV and grade III to IV aGVHD was 43% and 18% for MAC and 33% and 0% for RIC. 4. The 1-year incidence of chronic GVHD MAC and RIC was 36% and 18%, respectively. |
| Al Malki et al (2021)29 | 38 patients who underwent PBSC MMUD HCT | Single-center, prospective study | 1. The 1-year OS was 87%. 2. The 1-year GRFS 68%. 3. The day +100 incidence of grade II to IV and III to IV aGVHD was 50% and 18%, respectively. 4. The 1-year incidence of chronic GVHD was 48%. | |
| Battipaglia et al (2022)36 | Patients with AML in CR undergoing MMUD HCT (n = 155) compared to Haplo BM (n = 647) and Haplo PB (n = 949) with PTCy-based GVHD prophylaxis | Retrospective study | Haplo HCT | 1. Haplo BMSC and Haplo PBSC had a higher NRM compared to MMUD (HR, 2.28; 95% CI, 1.23-4.24; P < .01 and HR, 2.65; 95% CI, 1.46-4.81; P < .01, respectively) with lower LFS and OS. |
| Battipaglia et al (2019)37 | 272 patients with AML underwent 9/10 HLA matched HCT with GVHD prophylaxis consisting of PTCy-based (n = 93) or ATG-based (n = 179) regimens. HLA mismatch involved class I in 74% and class II in 26%. Half of the patients received MAC, and the other half received RIC. | Retrospective study | 1. Use of PTCy was associated with lower incidence of severe aGVHD and higher LFS and GRFS. | |
| Watkins et al (2021)40 | 38 received MMUD, mostly MAC, with 48% receiving PBSCs and 52% received BMSCs. GVHD prophylaxis with abatacept in combination with CNI and MTX | Phase 2 | Historical cohort from CIBMTR with or without ATG | 1. The incidence of grade II to IV aGVHD was 2.3% (CNI/MTX plus abatacept, intention-to-treat population), which compared favorably with a nonrandomized matched cohort of CNI/MTX (30.2%, P < .001), and the SGFS was better (97.7% vs 58.7%, P < .001). |
| Kean et al (2021)41 | 7/8 HLA MMUD HCTs for hematologic malignancies between 2011 and 2018 using either CNI + MTX with (n = 54) or without ABA (n = 162) | CIBMTR retrospective | 1. The OS at day +180 for the cohort receiving ABA was 98% compared to 75% for CNI + MTX alone (P = .0028). 2. In an exploratory analysis focused on short-term end point (OS at 180 days), outcome of patients undergoing MMUD HCT with ABA with CNI + MTX was comparable to patients undergoing PTCy. |
| Study . | Population . | Design . | Comparison . | Key findings . |
|---|---|---|---|---|
| Shaw et al (2021)35 | 80 patients who underwent RIC/MAC BMSC MMUD HCT with PTCY/MMF/sirolimus-based GVHD prophylaxis | Multicenter, prospective, phase 2 trial | NA | 1. The 1-year OS was 76% (72% for MAC and 79% for RIC) 2. Degree of HLA mismatch did not affect the OS (75% for 7/8 and 77% for 4-6/8). 3. The day +100 incidence of grade II to IV and grade III to IV aGVHD was 43% and 18% for MAC and 33% and 0% for RIC. 4. The 1-year incidence of chronic GVHD MAC and RIC was 36% and 18%, respectively. |
| Al Malki et al (2021)29 | 38 patients who underwent PBSC MMUD HCT | Single-center, prospective study | 1. The 1-year OS was 87%. 2. The 1-year GRFS 68%. 3. The day +100 incidence of grade II to IV and III to IV aGVHD was 50% and 18%, respectively. 4. The 1-year incidence of chronic GVHD was 48%. | |
| Battipaglia et al (2022)36 | Patients with AML in CR undergoing MMUD HCT (n = 155) compared to Haplo BM (n = 647) and Haplo PB (n = 949) with PTCy-based GVHD prophylaxis | Retrospective study | Haplo HCT | 1. Haplo BMSC and Haplo PBSC had a higher NRM compared to MMUD (HR, 2.28; 95% CI, 1.23-4.24; P < .01 and HR, 2.65; 95% CI, 1.46-4.81; P < .01, respectively) with lower LFS and OS. |
| Battipaglia et al (2019)37 | 272 patients with AML underwent 9/10 HLA matched HCT with GVHD prophylaxis consisting of PTCy-based (n = 93) or ATG-based (n = 179) regimens. HLA mismatch involved class I in 74% and class II in 26%. Half of the patients received MAC, and the other half received RIC. | Retrospective study | 1. Use of PTCy was associated with lower incidence of severe aGVHD and higher LFS and GRFS. | |
| Watkins et al (2021)40 | 38 received MMUD, mostly MAC, with 48% receiving PBSCs and 52% received BMSCs. GVHD prophylaxis with abatacept in combination with CNI and MTX | Phase 2 | Historical cohort from CIBMTR with or without ATG | 1. The incidence of grade II to IV aGVHD was 2.3% (CNI/MTX plus abatacept, intention-to-treat population), which compared favorably with a nonrandomized matched cohort of CNI/MTX (30.2%, P < .001), and the SGFS was better (97.7% vs 58.7%, P < .001). |
| Kean et al (2021)41 | 7/8 HLA MMUD HCTs for hematologic malignancies between 2011 and 2018 using either CNI + MTX with (n = 54) or without ABA (n = 162) | CIBMTR retrospective | 1. The OS at day +180 for the cohort receiving ABA was 98% compared to 75% for CNI + MTX alone (P = .0028). 2. In an exploratory analysis focused on short-term end point (OS at 180 days), outcome of patients undergoing MMUD HCT with ABA with CNI + MTX was comparable to patients undergoing PTCy. |
ATG, antithymocyte globulin; GRFS, GVHD free relapse-free survival; HR, hazard ratio; MMF, mycophenolate mofetil; SGFS, severe aGVHD-free survival.