Among patients with de novo acute myeloid leukemia (AML), fractionated-dose gemtuzumab ozogamicin (GO) does not appear to increase the risk of hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) post-transplant, according to research published in Bone Marrow Transplantation.

GO is a humanized anti-CD33 monoclonal antibody indicated for newly diagnosed, CD33-positive AML. While a previous phase 3 trial (ALFA-0701; Identifier: NCT00927498) showed that fractionated GO is both safe and effective when used with standard chemotherapy, VOD/SOS risk remained a concern among patients undergoing hematopoietic stem cell transplantation (HSCT).

There is, however, some evidence that fractionated GO dosing may mitigate the risk of VOD/SOS prior to HSCT. In this retrospective analysis, researchers evaluated the occurrence of VOD/SOS and post-transplant outcomes among patients who underwent HSCT as follow-up therapy in ALFA-0701.

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In ALFA-0701, researchers randomly assigned patients aged 50 to 70 years to receive either GO 3 mg/m2 with standard chemotherapy or standard chemotherapy only. A subset of patients (n=85) was then recommended to receive HSCT either in first complete remission or after induction failure/relapse. Of these patients, 32 were in the GO group, while 53 were in the standard chemotherapy group.

Overall, 3.5% of HSCT recipients (3/85) developed VOD/SOS (GO group, 2; standard chemotherapy group, 1). The 2 patients in the GO group developed VOD/SOS during induction and recovered, and 1 patient developed VOD/SOS after second HSCT.

Overall survival [OS] did not differ between arms in patients who received HSCT but no longer on GO (hazard ratio [HR], 0.97; 95% CI, 0.54-1.75; P =.919) vs patients who did not receive HSCT (HR, 0.68; 95% CI, 0.48-0.97; P =.0333).

“[T]hese findings suggest that fractionated-dose GO as part of induction and consolidation chemotherapy for adult AML does not induce excess post-transplant mortality and VOD/SOS and thus does not preclude the use of HSCT as consolidation treatment following induction or salvage treatment,” the authors concluded.

Disclosures: Some authors have declared affiliations with or received funding from the pharmaceutical industry. Please refer to the original study for a full list of disclosures.


Pautas C, Raffoux E, Lambert J, et al. Outcomes following hematopoietic stem cell transplantation in patients treated with standard chemotherapy with or without gemtuzumab ozogamicin for acute myeloid leukemia. Bone Marrow Transplant. Published online February 9, 2020. doi:10.1038/s41409-020-01207-4