Myeloproliferative Neoplasms: Treatment Approach and Outcomes
The Drexel Experience
Sukhdeep Kaur1, Courtney Ackerman, Hareesha Chakunta, Michael Styler1, Division of Hematology and Oncology, Department of Medicine, Hahnemann
University Hospital, Drexel University College of Medicine, Philadelphia, PA
INTRODUCTION
RESULTS
RESULTS
CYTOREDUCTION THERAPY
One hundred eighteen patients’ charts were reviewed and categorized based on treatment.
Myeloproliferative neoplasms are a group of clonal disorders that arise
from a transformation in a hematopoietic stem cell. When determining
treatment strategies for these patients, one must consider long-term
survival, morbidity from thrombotic complications, development of
myelofibrosis or transformation into acute leukemia, and the effect of
specific therapies on the incidence of leukemic transformation and on
pregnancy. At Drexel University, a significant number of patients were
treated with busulfan and were thought to have a more favorable clinical
course and possibly increased survival in comparison to other agents. In
our study we analyzed the outcomes of patients treated in the practice of I.
Brodsky Associates diagnosed with essential thrombocytosis (ET),
polycythemia vera (PCV), primary Myelofibrosis (PMF), and
Myeloproliferative disorder NOS, who received a variety of treatment
modalities, and compared their clinical courses to determine if there is a
superior treatment.
Percent survival
100
80
60
40
20
0
0
100
200
300
400
500
MONTHS
P. VERA
E. THROMBOCYTOSIS
HYDROXYUREA 1 (7)
HYDROXYUREA 2 (13)
BUSULFAN 1 (19)
BUSULFAN 2 (14)
PATIENTS AND METHODS
jGRAPH 1: Complications of groups based on treatment.
This study is a retrospective cohort study in which we examined the
medical records of patients treated for the diagnoses of ET, PCV and PMF
at Hahnemann Hospital-Drexel University College of Medicine in the
practice of I. Brodsky Associates from January 1960 to December 2013.
The following variables were measured and compared (Table 1):
Thrombotic events were most common in the Busulfan and Hydroxyurea combination group (56%). Second malignancy
was diagnosed in 31% of patients treated with Hydroxyurea alone. Leukemic transformation occurred in 19% of patients
treated with hydroxyurea alone. Hemorrhage occurred in 17% of patients in busulfan group. “Other” were considered to be
transfusion dependent anemia, progression to myelofibrosis, and development of varices without bleeding.
ESSENTIAL THROMBOCYTOSIS
100
E.
P.
Thrombocytosis Vera
Total number of Pts
47
Primary
Myelofibrosis
56
Myeloproliferative
d/o NOS
8
Percent survival
Baseline
characteristics
7
Age (years)
RANGE
MEDIAN
41-97 40-97
70
75
37-85
35-96
63
67
Sex
80
60
40
20
0
Male
16
35
4
5
Female
31
22
4
2
0
ASPIRIN (9)
51
6
4
African American
9
6
1
2
Hispanic
0
0
1
0
Asian
1
0
0
1
Hemoglobin
13.2
17.7
11.3
11.1
Hematocrit
39.6
53.1
36.5
34.2
Platelet (thousand)
968
424
301
173
WBC
8.5
10.2
13.7
12.8
Labs at dx (median)
jGRAPH
20
33
8
3
No
23
23
0
3
14
19
0
1
Hydroxyurea
13
7
3
3
Busulfan+Hydroxyurea
10
10
3
1
Aspirin/plavix only
9
14
2
0
Phlebotomy only
JAK 2 Mutation
documented
Chromosome
anomaly
2
7
1
2
HYDROXYUREA (13)
5
12
6
BUSULFAN (14)
2: Overall Survival by Treatment Group in Essential Thrombocytosis
100
80
60
20
0
100
200
300
400
500
MONTHS
ASPIRIN (13)
HYDROXYUREA (7)
0
REFERENCES
BUSULFAN (19)
PHLEBOTOMY (7)
1
3
Our retrospective study showed that busulfan used alone showed longest
survival as well as having less complications. The significant amount of
complications from thrombotic events noted in the dual treatment with busulfan
and hydroxyurea occurred mostly prior to starting treatment with busulfan. Given
longest median survival in this group, busulfan may provide the optimal disease
control in patients with myeloproliferative neoplasms, and thus most effectively
reduce the risk of thrombosis. Moreover, increased risk of second malignancies,
including transformation to acute leukemia, was not seen in the busulfan treated
patients. This suggests that physicians should consider the use of busulfan in
treating myeloproliferative neoplasms.
40
0
jGRAPH
DISCUSSION
PHLEBOTOMY (1)
BUSULFAN/HYDROXYUREA (10)
9
500
POLYCYTHEMIA VERA
Treatment
Busulfan
400
Patients that were treated with a combination of Busulfan and Hydroxyurea had the longest survival of 398 months, while
Busulfan alone had the longest survival of 455 months,
Splenomegaly
Yes
300
BUSULFAN/HYDROXYUREA (10)
Percent survival
37
200
MONTHS
Ethnicity
Caucasian
100
3: Overall Survival by Treatment Group in Polycythemia Vera
Patients that were treated with combination of Busulfan and Hydroxyurea had the longest survival of 492 months, while
Busulfan alone had the longest survival of 457 months,
1.  Zittoun R, the EORTC Leukemia and Haematosarcoma Cooperative Group. Busulfan versus 32P in
polycytheaemia vera. Drugs Exp Clin Res 1986;12:283-6
2.  Brodsky I. Busulphan treatment of polycythemia vera. Br J Haematol 1982;52
3.  :1-6.