Blood journal snippets

 Blood journal Feb 2025

1.  Dual Asciminb and dasatinib inPh+ ALL and CML blast crisis:  80 mg asciminb+ dasatinib 140 mg daily  plus prednisone 60 mg/m2 for 28 days--> CHR84%. By day 84--> 100% complete hematologic response. After 28 day induction, the combination TKI was continued until transplant. Pancreatic enzyme elevation without pancreatitis was a toxicity. No vasoocclusion.

2. Blastic plasmacytoid dendritic neoplasm

- an aggressive heme malignancy derived from plasma dendritic cell

-IHC--> CD 123+, CD4 or CD56+, plus one other plasma cell dendritic cell marker CD303 or CD304. TET 2 mutations are common

- Mature dendritic cells lose CD56 which is not the case with BPDCN ( CD 56 positive)

-less than 0.5% incidence

-most common organ affected --> skin. CNS involvement can be 2 to 60%.

-CSF at baseline recommended for all patients with BPDCN regardless of CNS symptoms

-Ddx- AML with monocytic differentiation, CMML

-Rx--Induction with antiCD123 drug conjugate tagraxofusp ( Human IL-3 + truncated diphtheria toxin)

- Allo HCT in CR1; auto SCT can be considered in elderly IF no bone marrow involvement at presentation.

- even if pt has a localized skin presentation, XRT alone is futile and systemic chemo with allo HCT should be the goal

-front line options: none standard. Tagraxofusp or Venetoclax+ hyper CVAD, or ven+ Aza

From Open Evidence.

The treatment for blastic plasmacytoid dendritic cell neoplasm (BPDCN) has evolved significantly in recent years. The current first-line treatment for BPDCN is tagraxofusp, a CD123-targeted therapy. Tagraxofusp is a recombinant fusion protein that combines interleukin-3 with a truncated diphtheria toxin, and it has shown a high overall response rate in clinical trials. The typical dosage of tagraxofusp is 12 µg/kg administered intravenously on days 1 to 5 of a 21-day cycle.

For patients who achieve complete remission (CR) with tagraxofusp, allogeneic hematopoietic stem cell transplantation (allo-HCT) is recommended as a consolidative therapy, as it offers the best chance for long-term survival.[1][4] The National Comprehensive Cancer Network (NCCN) guidelines emphasize the importance of achieving CR before proceeding to allo-HCT.

In cases of relapsed or refractory BPDCN, treatment options include re-administration of tagraxofusp (if not previously used), chemotherapy, local radiation to isolated lesions, systemic steroids, and venetoclax-based regimens. Participation in clinical trials is also highly encouraged for these patients.

Close monitoring for adverse effects, particularly capillary leak syndrome, is crucial during tagraxofusp therapy.