ASH Clinical News Advances in Hematology Research & Patient Care: Hi | Page 17
TABLE 3. Quality-of-Life Comparison for Eltrombopag Versus Placebo at Baseline and 12 Weeks Post-Treatment
Eltrombopag
Quality-of-Life Measure
Placebo
Baseline
(median, out of 100)
12 Weeks
(median, out of 100)
Baseline
(median, out of 100)
12 Weeks
(median, out of 100)
Physical
50
62
62
37
Functional
50
33
61
44
Social
50
50
50
38
Sexual
63
75
67
83
Fatigue
71
76
69
76
MDS-specific
67
71
56
69
Treatment outcome index
56
56
56
47
counts <30 x 109/L and had an Eastern Cooperative Oncology Group
performance status of <4. The mean patient age was 68.3 years, and 38
percent of participants were male.
In patients with baseline platelet count >20 x 109/L, response
was defined as absence of bleeding and an increase in platelets of
≥30 x 109/L from baseline. In patients with baseline platelet count
<20 x 109/L, response was defined as a platelet increase to >20 x
109/L and an increase by at least 100 percent (not due to platelet
transfusion). Complete response was defined as a platelet count
≥100 x109/L and an absence of bleeding.
At the time of this interim analysis, 23 patients (50%) receiving eltrombopag had responded to treatment, compared with two
patients (8%) receiving placebo (p=0.016). The median time to
response was 14 days, and the median dose was 75 mg.
The platelet count increased by a mean of 53.2 x 109/L in patients receiving eltrombopag, while patients in the placebo cohort
have demonstrated no significant change in platelet counts by 24
weeks of treatment. Eltrombopag treatment also improved several
measures of quality of life, according to responses to a questionnaire
evaluating MDS-specific symptom burden (TABLE 3).
Eltrombopag appeared to be well tolerated, with patients experiencing the following treatment-related grade 3 or 4 adverse events:
• Nausea (n=4)
• Hypertransaminasemia (n=3)
• Hyperbilirubinemia (n=1)
• Sepsis (n=1)
• Pruritus (n=1)
• Heart failure (n=1)
• Asthenia (n=1)
• Vomiting (n=1)
One patient in the placebo group experienced grade 3 bone marrow
fibrosis.
“The drug appears to be well tolerated and not significantly
associated with MDS progression,” Dr. Oliva reported, though five
eltrombopag-treated patients (11%) experienced MDS disease progression, compared with two patients in the placebo group (8%).
“Preliminary data indicate that lower-risk MDS patients with severe
thrombocytopenia undergoing treatment with eltrombopag experience
significant improvements in platelet counts accompanied by improvements in fatigue,” Dr. Oliva and colleagues concluded. However, further
follow-up is required to evaluate the effect on survival, she added.
Reference
Oliva EN, Santini V, Alati C, et al. Eltrombopag for the treatment of
thrombocytopenia of low and intermediate-1 IPSS risk myelodysplastic syndromes:
interim results on efficacy, safety and quality of life of an international, multicenter
prospective, randomized trial. Abstract #91. Presented at the ASH Annual Meeting,
December 5, 2015; Orlando, Florida.
Early PET Scans Can Safely
Guide Chemotherapy DeEscalation in Patients with
Hodgkin Lymphoma
Although escalated BEACOPP (bleomycin, etoposide, doxorubicin, cyclophosphamide, vincristine, procarbazine, and
prednisone), or BEACOPPesc, has been shown to be superior to ABVD chemotherapy in controlling disease in patients
with advanced-stage Hodgkin lymphoma, it is also associated with higher toxicity, including immediate hematologic
toxicity and an increased risk of secondary myelodysplasia
or leukemia and infertility.
Given these risks, identifying patients with HL who respond early
to BEACOPPesc would allow physicians to begin a strategy of
de-escalating treatment intensity – hopefully without impairing the
disease control.
In a study presented last month at the ASH annual meeting,
Olivier Casanovas, MD, from the Hematology Department at
January 2016
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