TRAINING and EDUCATION
You Make the Call
Each month in “You Make the Call,” we’ll pick a challenging clinical question
submitted through the Consult-a-Colleague program and post the expert’s
response. But, what would YOU do? We’ll also pose a submitted question
and ask you to send your responses. See how your answer matches up to the
experts’ in the next print issue.
This month, Richard A. Larson, MD, answers a question about the treatment
of blastic plasmacytoid dendritic cell neoplasm.
Clinical Dilemma:
The patient is a 73-year-old man with blastic plasmacytoid dendritic cell neoplasm and a long history of anemia. A bone
marrow biopsy three years ago showed only iron deficiency and a gastrointestinal evaluation was unremarkable. The
patient received iron replacement therapy, but his anemia never improved. He now presents with purple skin lesions.
A biopsy showed blastic plasmacytoid dendritic cell neoplasm, and a bone marrow biopsy showed very cellular bone
marrow with 60 to 70 percent blastic plasmacytoid dendritic cell neoplasm. Results from a cytogenetic analysis are pending. Meanwhile, he has been relatively asymptomatic. He is active and healthy and his blood count has been stable for the
last three years (white blood cell count is 3.3 x 109/L, hemoglobin count is 10.3 g/dl, and platelet count is 135 x 109/L). He
is also worried about toxicities with acute lymphocytic leukemia (ALL)–type treatment.
How would you treat this patient? What result can you expect from treatment?
Consult a Colleague
Through ASH
Consult a Colleague is a service for ASH
members that helps facilitate the exchange of information between hematologists and their peers. ASH members
can seek consultation on clinical cases
from qualified experts in 11 categories:
• Anemias
• Hematopoietic cell
transplantation
• Hemoglobinopathies
• Hemostasis/thrombosis
• Lymphomas
Experts Make the Call
Richard A. Larson, MD
Professor of Medicine
University of Chicago
Chicago, Illinois
This is a very uncommon disorder. The disease itself
can have a variable rate of progression, but it generally
leads to bone marrow failure and death. The malignant
cells are typically resistant to usual leukemia or lymphoma chemotherapy regimens; although remissions
can be achieved, they are short in duration.
The majority of patients who have achieved longterm survival have had an allogeneic transplant, usually
early in first remission, and children have better outcomes than older patients.
Typically, we initiate therapy with an ALL regimen appropriate for patients of the same age. For your
patient, this might include vincristine, dexamethasone,
and daunorubicin, with or without cyclophosphamide.
Many older patients do not tolerate asparaginase
well, which we prefer to use in younger patients with
ALL. If the initial treatment is unsuccessful, then we
often switch to a high-dose cytarabine regimen. Others
use a chemotherapeutic AML regimen such as 7+3. I
am not aware of therapy with azacitidine or decitabine
for blastic plasmacytoid dendritic cell neoplasm, but I
50
ASH Clinical News
• Leukemias
• Multiple myeloma & Waldenström
macroglobulinemia
• Myeloproliferative Disorders
• Myelodysplastic Syndromes
• Thrombocytopenias
suspect this approach has been tried.
There also may be a clinical trial available to your
patient. Recently, there were some encouraging data published in Blood about an anti-CD123 immunotoxin in this
disease, which represented the first prospective study of
treatment of patients with blastic plasmacytoid dendritic
cell neoplasm.1 In this study, 11 patients were treated with
a single course of SL-401 at 12.5 μg/kg intravenously over
15 minutes daily for up to five doses; three patients who
had initial responses to SL-401 received a second course
in relapse. Seven of the nine evaluable (78%) patients had
major responses after a single course of SL-401, and the
median duration of responses was five months. The most
common adverse events including fever, chills, hypotension, edema, hypoalbuminemia, thrombocytopenia, and
transaminasemia were transient. ●
REFERENCE
Frankel AE, Woo JH, Ahn C, et al. Activity of SL-401, a targeted therapy directed to interleukin-3
receptor, in blastic plasmacytoid dendritic cell neoplasm patients. Blood. 2014 Jul;124:385-92.
Next Month’s Clinical Dilemma:
I have seen several orthopedic surgeons
give tranexamic acid before and during
surgery. One surgeon did not even hold
the warfarin? Is this the standard now?
• Lymphoproliferative disorders
How would you respond? Email us at
[email protected].
Assigned volunteers (“colleagues”) will
respond to inquiries within two business
days (either by email or phone).
Have a puzzling clinical dilemma?
Submit a question, and read more
about Consult-a-Colleague volunteers at
hematology.org/Clinicians/Consult.aspx
or scan the QR code.
*If you have a request related to a
hematologic disorder not listed here, please
email your recommendation to ashconsult@
hematology.org so it can be considered for
addition in the future.
DISCLAIMER: ASH does not recommend
or endorse any specific tests, physicians,
products, procedures, or opinions, and
disclaims any representation, warranty, or
guaranty as to the same. Reliance on any
information provided in this article is solely
at your own risk.
October 2015