A timeline to transplant that usually takes months took almost two years for patient with a “poor risk” diagnosis.
For one 60-year-old leukemia patient at USC Norris Comprehensive Cancer Center, her oncologists knew from the start that a myeloablative allogenic stem cell transplant (bone marrow transplant) offered the best chance of a long-term cure.
The patient’s diagnosis indicated “poor risk” AML with RUNX1, ASXL1, NRAS and EZH2 mutations. So, in order to give her the best outcome after transplant, USC Norris doctors first needed to get her into remission with 7 + 3 chemotherapy followed by consolidation with high-dose cytarabine (HiDAC).
This regimen put the patient into a brief remission, but she quickly relapsed before she could undergo a bone marrow transplant.
“She got into remission with initial treatment, but she relapsed,” said Eric Tam, MD, a specialist in hematology and blood and marrow transplant at USC Norris, part of Keck Medicine of USC.
Working to achieve remission
The USC Norris team worked to find an alternate route to ensure the patient could once again go into remission and safely endure a transplant.
When a bone-marrow biopsy revealed some residual disease in the patient, Tam administered an oral hypomethylating agent (HMA) in combination with venetoclax, a type of chemotherapy used when patients cannot endure more intensive chemotherapy, or when they have a high risk of resistance to chemotherapy.
During the course of her treatment, the patient developed Sweet’s syndrome on one hand, her chest and a leg, which was treated with steroids.
Still, the regimen wasn’t enough. Repeated bone marrow biopsies revealed that the patient continued to have a low level of residual disease.
“We were having a hard time getting her back into remission, even though we tried several different regimens over the course of a year,” Tam said. “She was even on a clinical trial at one point.”
Persistent treatment supports bone marrow transplant
The care team at USC Norris left no stone unturned in the patient’s journey to transplant, which, in this case, was longer than expected. That’s because leukemia patients usually receive bone marrow transplants within months of their diagnosis.
For this patient, due to her cancer’s persistence and resistance to treatment, it took almost two years.
After a round of cytotoxic chemotherapy with FLAG + venetoclax chemotherapy gave no additional response, she continued decitabine + venetoclax until she registered just .05% disease.
She then received a bone marrow transplant in March 2022, and is currently in the recovery phase, tapering off immunosuppressants and preparing to return to work as a paralegal.
The USC Norris doctors are optimistic about the road ahead. “So far, she’s been doing well with minimal side effects and minimal complications,” said Tam. “And there’s no evidence of leukemia or GVHD.”
A leading center for BMT survival rates
The USC Norris Blood and Marrow Transplant and Cell Therapy Program is one of the top transplant centers in the country. While the national average for the one-year survival rate of adult allogeneic bone marrow transplants is 76.5%, the rate for USC Norris patients is 90.3%, according to the Center for International Blood and Marrow Transplant Research, which tracks the statistic annually.
This survival rate is among the highest in the nation and follows many years of a one-year survival rate of 80% to 88%. In addition, the USC Norris program was one of only 11 U.S. transplant centers that performed better than expected and is the only center that beat its expected survival rate by double digits.