CASE:
15 years old, male patient was diagnosed BPDCN in January 2019 with
generalized lymphadenopathy and maculopapular skin rash on trunk. AML
BFM 2013 was given but one month after last cycle, he relapsed with
macular rash. BPDCN was confirmed, Patient was treated with two cycles
of FLAG-IDA and a cycle of Hyper-CYVAD regimens, no remission obtained.
Tagraxofusp-erzs was planned but patient had COVID-19 pneumonia with no
worsening of his ECOG score. Venetoclax initiated 100 mg/day and
increased to 400 mg/day in a week, no toxicity and no tumor lysis
syndrome was occurred. In combination first cycle of tagraxofusp- erz
was given at a dose of 12 mcg/kg/day/5 days. A reversible moderate
hepatotoxicity and myelosuppression occurred. There was no sign of
capillary leak syndrome which is the most serious and not so rare side
effect. On the second day of second cycle, capillary leak syndrome was
diagnosed with sudden weight gain, fluid retention, hypoalbuminemia and
slight hypotension. Therapy was ceased. Patient recovered with the
supportive treatment including inotropic agent within 5 days. Responses
after chemotherapy and side effects are outlined at Table -1 and
supplemental Table S1.
The patient admitted to stem cell transplantation unit on July 2020 with
remission. Myeloablative regimen was busulfan, melphalan and
cyclophosphamide. Neutrophile engraftment occured on 10 th day of
transplant. He followed for six months in remission. At 6 th month of
transplant BPDCN blasts were positive for both bone marrow and central
nervous system. Patient was considered ineligible for intensive
chemotherapy and repeated tagraxofusp-erz due to lack of experience in
relapsed cases and previous capillary leak syndrome side effect.
Azacitidine plus venetoclax combination which was shown to be effective
in AML for elderly (3) was considered safe rather than standard
chemotherapy. Azacitidine was given 100 mg/m2/day for five days sc and
oral venetoclax 400 mg/day. He had no response and died.