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, Marcie Riches, MD, MS, answers a question about stem cell
transplantation in a patient who presents with aplastic anemia and PNH.
Clinical Dilemma:
A 28-year-old patient presented with aplastic anemia (AA) (diagnosed at age 15) and paroxysmal nocturnal hemoglobinuria (PNH). A bone marrow biopsy showed 15 perce nt cellularity. He responded to cyclosporine and ATG but
was lost to follow-up. When he came back about eight years ago, he was noted to have pancytopenia with hemolysis
and a PNH clone was found, representing 12.4 percent of his total RBCs. He was prescribed eculizumab and though
improvement was observed, blood counts did not normalize. His last bone marrow biopsy was normocellular. Is there
a role for stem cell transplantation in this patient?
Experts Make the Call
Marcie Riches, MD, MS
Associate Professor
Director of Clinical Research and Data Quality
University of North Carolina Bone Marrow and Stem
Cell Transplant Clinic
Chapel Hill, North Carolina
This is an interesting case. I have some questions that
are relevant to my response regarding the role of stem
cell transplantation.
As I understand from your case, this is a 28-year-old
male who at 15 years old had AA (15% cellular bone
marrow) treated with CSA and ATG. He was lost to
follow-up for five years and resurfaced eight years ago
with pancytopenia and a small PNH clone.
1. How severe was his pancytopenia at re-presentation?
A: 2010: WBC 2.6, HGB 8, Plts 55K, ANC 1.5;
2015: WBC 3.6, HGB ~10, Plts 118, ANC 2K.
5. Are there any additional cytogenetic abnormalities
noted or another potential etiology for the
cytopenias?
A: None on multiple bone marrow
biopsies.
6. Is there any pertinent family history
of marrow dysfunction?
A: Unknown. Both parents died young
in an accident, and there is no other
family history.
Given that the patient is transfusionindependent, it is reasonable to start
the donor search, but with an ANC
2 and plts >50K, it’s also reasonable to hold off for now until
counts worsen.
Next Month’s Clinical Dilemma:
A 44-year-old male with no significant
past medical history presented after
feeling supraclavicular adenopathy on
a self-exam. His physician ordered a
biopsy, which showed Hodgkin lymphoma, nodular sclerosis subtype. A CT
showed neck and hilar adenopathy, with
a maximum size of 2-3 cm. The PET scan
showed supraclavicular and mediastinal
bone disease and right hilar adenopathy,
as described above, as well as osseous
metastatic disease with lesions in the
right humeral head, thoracic and lumbar
spine, left ribs, and left ilium. Would
you recommend a bone marrow biopsy
to complete staging? Would you give GCSF, given the conflicting evidence about
the associated bleomycin lung toxicity?
How would you respond? Email us at
[email protected].
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
2. How long has the patient been receiving Solaris?
A: Five years, but the patient has periods without
treatment due to insurance issues.
• Lymphomas
3. Is the patient transfusion independent?
A: Yes.
• Multiple myeloma & Waldenström
macroglobulinemia
4. What is the current % PNH?
A: Similar to prior PNH clone size.
• Lymphoproliferative disorders
• Leukemias
• Myeloproliferative Disorders
• Myelodysplastic Syndromes
• Thrombocytopenias
Assigned volunteers (“colleagues”) will
respond to inquiries within two business
days (either by email or phone).
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.
64
ASH Clinical News
Have a puzzling clinical dilemma?
Submit a question, and read more
about Consult-a-Colleague volunteers at
hematology.org/Clinicians/Consult.aspx.
February 2016