What the trial was testing
The trial enrolled 87 patients with lymphoma. The study was sponsored by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was an early-stage trial — researchers are still confirming safety and getting an early look at how well the treatment works. Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
75% alive at 1 year — and no severe acute graft-vs-host disease.
Blood Advances · 2017 · NCT01203722
These findings — that at 1 year after mismatched unrelated donor bone marrow transplant with PTCy — were published in the Blood Advances and represent the headline result of the study.
Researchers tracked outcomes across 87 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with lymphoma, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Mismatched unrelated donor transplant with post-transplant cyclophosphamide is now offered at major bone marrow transplant centers as an option for patients who lack a matched donor. Ask a transplant hematologist about donor search and transplant options.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open lymphoma trials
Multicenter HomeVENT: Home Values and Experiences Navigation Track
This is a multicenter study to test a decision-making support process for families and clinicians facing decisions about chronic home ventilation for a child. The investigators hypothesize that the intervention will increase family preparedness for decision-making and will improve clinician-family shared-decision making. Half of families will be assigned to "usual care" arm and half to the "intervention" arm. Intervention families will view the study website with study staff and will answer questions related to website content. All families will be interviewed and surveyed at 1, 6 and 12 months after enrollment. Each family will designate 1-2 physician involved in the decision about home ventilation; each physician will be interviewed and surveyed at 1 month.
Use of Levocarnitine to Reduce Asparaginase Hepatotoxicity in Patients With Acute Lymphoblastic Leukemia
Acute lymphoblastic leukemia (ALL) is the most common cancer seen in pediatric oncology. The necessary chemotherapy for pediatric and adolescent and young adult (AYA) patients with ALL includes steroids, anthracyclines, asparaginase, and vincristine. One of the most hepatotoxic chemotherapy agents is asparaginase, with treatment-associated hepatotoxicity (TAH) observed in up to 60% of patients. The frequency of TAH is increased in overweight or obese patients of Latino heritage. Carnitine is a naturally-derived compound that is produced in the liver and kidneys; it is found in certain foods, such as meat, poultry, fish, and some dairy products. Endogenous carnitine transports long-chain fatty acids into the mitochondria, where they are oxidized to produce energy, and acts as scavengers of oxygen free radicals. Thus, carnitine can reduce oxidative stress and modulate inflammatory response. Levocarnitine is a supplement form of carnitine used typically in the care and management of patients with carnitine deficiency. Pediatric and AYAs with ALL will be given oral levocarnitine as a supplement during their initial phases of treatment, when the most hepatotoxic agents are administered, to determine if the incidence of liver toxicity can be reduced or eliminated.