Contributions
Abstract: PB1426
Type: Publication Only
Session title: Acute myeloid leukemia - Clinical
Background
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare myeloid malignancy, for which conventional chemotherapy has poor outcomes. Tagraxofusp (SL-401) is a CD123-directed cytotoxin consisting of human interleukin-3 fused to truncated diphtheria toxin which has recently been approved by EMA.
Aims
To describe our positive experience with Tagraxofusp and to present our diagnosis process and observed adverse events.
Methods
We present the case of a 38-year-old female, who presented with pancytopenia, polyadenopathy and skin lesions and was diagnosed with blastic plasmacytoid dendritic cell neoplasia with complex karyotype, PTPN11 mutation by NGS Expression. Infiltration of bone marrow, skin, lymph nodes and CNS was noted.
We administered Tagroxofusp according to the dose of the technical data with 12 mcg/kg by intravenous infusion over 15 minutes, once a day, on days 1-5 of a 21-day cycle considering that this administration could be extended with delays until day 10 of the cycle. Prior to preparing each dose vital signs, albumin, transaminases, creatinine, body weight, sistolic blood pressure and heart rate was made.Treatment was administered as a bridge therapy to allogeneic hematopoietic stem cell transplantation (HSCT)
Results
Until the availability of the drug; 4 doses of intrathecal treatment were administered. Two weeks after diagnosis, treatment with tagraxofusp was started. During this time the patient maintained a good general condition. After the second dose of tagraxofusp a grade 2 capillary release syndrome (CLS) emerged. Other adverse events were shaking chills, rash, weight gain, and hepatotoxicity. After resolution of these problems, the next dose of treatment was administered after 21 days with a total of 3 cycles with excellent tolerance in a total of 5 days and without observing adverse events. Evaluation of the disease showed complete remission (medullary, skin, peripheral, CNS, and lymph node) with positive MRD (0.09). The 4th cycle remains pending prior to HSCT.
Conclusion
The use of tagraxofusp specially as a front-line therapy has shown in this patient promising clinical activity, with a high overall response rate and prolonged responses and acceptable toxicity. CLS grade 2 was observed only in cycle 1, therefore can be good therapeutic option for this hematological disease. Nevertheless future work will define its place in treating relapse or refractory disease as well as its role as a first line therapy or bridge to transplantation.
Keyword(s):
Abstract: PB1426
Type: Publication Only
Session title: Acute myeloid leukemia - Clinical
Background
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare myeloid malignancy, for which conventional chemotherapy has poor outcomes. Tagraxofusp (SL-401) is a CD123-directed cytotoxin consisting of human interleukin-3 fused to truncated diphtheria toxin which has recently been approved by EMA.
Aims
To describe our positive experience with Tagraxofusp and to present our diagnosis process and observed adverse events.
Methods
We present the case of a 38-year-old female, who presented with pancytopenia, polyadenopathy and skin lesions and was diagnosed with blastic plasmacytoid dendritic cell neoplasia with complex karyotype, PTPN11 mutation by NGS Expression. Infiltration of bone marrow, skin, lymph nodes and CNS was noted.
We administered Tagroxofusp according to the dose of the technical data with 12 mcg/kg by intravenous infusion over 15 minutes, once a day, on days 1-5 of a 21-day cycle considering that this administration could be extended with delays until day 10 of the cycle. Prior to preparing each dose vital signs, albumin, transaminases, creatinine, body weight, sistolic blood pressure and heart rate was made.Treatment was administered as a bridge therapy to allogeneic hematopoietic stem cell transplantation (HSCT)
Results
Until the availability of the drug; 4 doses of intrathecal treatment were administered. Two weeks after diagnosis, treatment with tagraxofusp was started. During this time the patient maintained a good general condition. After the second dose of tagraxofusp a grade 2 capillary release syndrome (CLS) emerged. Other adverse events were shaking chills, rash, weight gain, and hepatotoxicity. After resolution of these problems, the next dose of treatment was administered after 21 days with a total of 3 cycles with excellent tolerance in a total of 5 days and without observing adverse events. Evaluation of the disease showed complete remission (medullary, skin, peripheral, CNS, and lymph node) with positive MRD (0.09). The 4th cycle remains pending prior to HSCT.
Conclusion
The use of tagraxofusp specially as a front-line therapy has shown in this patient promising clinical activity, with a high overall response rate and prolonged responses and acceptable toxicity. CLS grade 2 was observed only in cycle 1, therefore can be good therapeutic option for this hematological disease. Nevertheless future work will define its place in treating relapse or refractory disease as well as its role as a first line therapy or bridge to transplantation.
Keyword(s):