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LymphomaApril 2019Summary reviewed July 2026

A Targeted Injection Put Rare Blood Cancer in Remission in 72% of Untreated Patients

Researchers tested tagraxofusp, a targeted treatment, in 47 adults with blastic plasmacytoid dendritic-cell neoplasm (BPDCN), a rare and aggressive blood cancer. In previously untreated patients, 72% achieved complete or near-complete remission, and 45% were able to proceed to stem-cell transplant.

What the trial was testing

The trial enrolled 138 patients with lymphoma. The study was sponsored by Stemline Therapeutics, Inc. 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

72% of previously untreated patients achieved complete or near-complete remission with tagraxofusp.

The New England journal of medicine · 2019 · NCT02113982

These findings — that achieved complete or near-complete remission — were published in the The New England journal of medicine and represent the headline result of the study.

Researchers tracked outcomes across 138 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

Tagraxofusp received FDA approval for BPDCN in December 2018 and is now available as a treatment option. The drug works by targeting a protein that BPDCN cells overexpress. Serious side effects including capillary leak syndrome and liver problems can occur, so close monitoring is required. Talk to your doctor about whether this treatment is right for you.

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.