12th Congress of the European Hematology ... - Haematologica

the therapy of choice for POEMS is APBSCT and that it should be performed
as front-line therapy in order to reduce risks of APBSC due to the
progressive organ damage. In this study 4 patients affected by POEMS
syndrome were treated with high dose chemotherapy and autologous
peripheral stem cell transplantation (aPBSCT). Three patients were male
and one female, median age was 53 yrs (44-62). At diagnosis all patients
had a severe, rapidly progressive sensory-motor peripheral neuropathy,
involving extremities, with inability to walk. All patients had melanosis,
monoclonal component IgA-lambda and 1 had also monoclonal component
IgG-lambda. Bone marrow biopsy documented in all patients mild
plasmacytosis (8-10%) endocrinopathy as thyropaty was present in all
patients and two patients experienced respectively hypogonadotropic
hypogonadism and hypophysary adenoma also. Two patient had
splenomegaly, and 2 hepatomegaly. One patient had sclerotic bone
lesion. Two patients were previously treated with high dose of intravenous
immunoglobulin. and steroids in the neurologic unit. One patient
had significantly low pulmonary function before aPBSCT. As induction/mobilization
therapy all patients received intermediate dose of
cyclophosphamide (1500 mg/m 2 on day 1,3) and Methylprednisolone
(250 mg from day 1-4) for 2 cycles. G-CSF was added after the 2nd cycle
in order to mobilizing peripheral stem cell. Time from diagnosis to aPB-
SCT was 5 months. Conditioning regimen was HDMel (Melphalan 100
mg/m 2 for 2 consecutive days). The median number of CD34 + cells
infused was 4.47 (range 3.08-5.63)×10 6 /kg. Engraftment was rapid and
sustained. After a median follow-up of 25.5 months (range 4-37), all
patients are alive with slow but progressive improvement in neurological
disease, skin changes, performance status and without evidence of
plasmacytosis. Organomegaly was resolved in both cases. Negativization
of monoclonal component was observed in a patient. Patient with
sclerotic bone lesion received radiotherapy (dose 5000 cGy) as consolidation.
Our experience confirms that HD-Mel and aPBSCT is feasible
and efficacious and should be the treatment of choice for POEMS, arresting
and even reversing the disease course. Early diagnosis is important
to led APBSCT that is able to obtain the best response and improve clinical
outcome.
0677
THE USE OF BORTEZOMIB AS FIRST LINE TREATMENT FOR PRIMARY PLASMA CELL
LEUKEMIA
G. Pietrantuono, 1 R. Guariglia,1 O. Villani, 1 F. D'Auria, 1 V. Pitini, 2
F. Rossini, 3 N. Filardi, 4 P. Musto1 1 Centro Riferimento Oncologico Basilicata, RIONERO IN VULTURE; 2 Medical
Oncology, University of Messina, MESSINA; 3 Hematology, S. Gerardo
Hospital, MONZA; 4 Hematology, S. Carlo Hospital, POTENZA, Italy
Background. We have recently shown that bortezomib is an effective
agent for the treatment of plasma cell leukemia (PCL), an aggressive,
rare variant of multiple myeloma (MM) (Musto et al, Cancer 2007, in
press). PCL represents about 2-4% of all MM and exists in two forms:
primary PCL (about 60% of cases) presents de novo in patients without
previous evidence of MM, while secondary PCL, which accounts for
the remaining 40%, consists of a leukemic transformation occurring in
about 1% of patients with a previously diagnosed MM. Aims. Our previous
study included both primary and secondary PCL, the majority of
whom were heavily pre-treated before of receiving bortezomib. In the
present study we focused on the effects of bortezomib as first line therapy
in primary PCL. Methods. Four patients (two male, two female; 61
to 76 years old) are so far evaluable. Circulating plasma cells ranged from
6 to 40×10 9 /L. Median WBC count was 40×10 9 /L (range 19-81). Two
patients had concomitant extramedullary disease (muscle and pleural
effusion). Del 13 was observed in 2 out of 3 patients with available karyotype.
Bortezomib was given using the standard schedule of 1.3 mg/sqm
days 1, 4, 8, 11, with an interval of 10 days between cycles. One patient
received dexamethasone and thalidomide, two doxorubicin and dexamethasone
(PAD) and one oral melphalan and prednisone (MPV) in combination
with bortezomib for 2-6 cycles. One patient underwent autologous
stem cell transplantation after 4 PAD cycles. Results. According to
the international uniform response criteria, three partial remissions
(reduction of M-component > 50%) and one very good partial remission
(disappearance of M-component at electrophoresis, but positive
immunofixation) were achieved (100% overall response). All patients are
alive after a mean follow-up of 8 months, without circulating plasma
cells in peripheral blood. Three out of them remain in remission phase,
one developed extramedullary progressive disease after 6 months. Grade
3-4 hematological toxicity and infections occurred in 2 patients. No other
significant adverse effects were observed. Summary. Global response
rate to standard chemotherapy in primary PCL is less than 50% and
12 th Congress of the European Hematology Association
median survival is only 7 months. Stem cell transplantation may be more
effective in some but not all patients. Our findings suggest that the frontline
use of bortezomib in combination with other active drugs is very
promising and could significantly improve the otherwise expected poor
clinical outcome of primary PCL Updated data about these and other not
yet evaluable patients will be presented.
0678
PRESENTATION AND SURVIVAL OF MULTIPLE MYELOMA PATIENTS: SIX YEAR SURVEY IN
A DISTRICT GENERAL HOSPITAL
A.S. Eswedi, R. Deore, M. Bowers, Y.L. Ong, C. McCoy, M. El-Agnaf
Ulster Hospital, BELFAST, United Kingdom
Background. Multiple Myeloma has an annual incidence of 5 in 100,000
populations. It has a wide spectrum of clinical features ranging from
asymptomatic paraproteinaemia to a rapidly progressive disease with
multiple end organ damage. Aims. To assess Multiple Myeloma presentation,
survival and therapy in a district general hospital. Methods. Retrospective
analysis of our databases for patients diagnosed with Multiple
Myeloma between 2000 and 2006. We were able to retrieve 60 out
of 69 patient records. Out of these 60 patients, 37 were males (61.7%)
and 23 were females (38.3%). Median age at presentation was 71 years
(36 to 89). Results. 24 patients (40%)were asymptmatic and were referred
because of incidental finding of paraproteinaemia. 3 patients (5%) progressed
from previous MGUS. The remaining 33 patients (55%) who
were symptomatic presented with: bone pain in 13 patients (21.7%),
Anaemia in 9 patients (15%), Renal failure in 3 patients (5%), weight loss
in 3 patients (5%), pancytopenia in 2 patients (3.3%), hypercalcaemia in
1 patient (1.7%), we also encountered a rare presentation as amyloidosis
in 1 patient (1.7%), and cord compression in another (1.7%). 48
patients (80%) were treated at presentation. The remaining 12 patients
(20%) were stable and did not require treatment. Of those who required
treatment; 27 patients (56.3%) were over 70 years of age and received
Melphalan with or without Prednisolone as a first line therapy. Patients
who were less than 70 years old received treatment in the form of: VAD
regimen in 4 (8.3%), Z-DEX in 10 (20.8%), Dexamethasone in 3 (6.3%),
Cyclophosphamide in 1 (2.1%), ABCM in 1 (2.1%), Prednisolone in 1
(2.1%) and Thalidomide in 1 (2.1%).Some patients received additional
treatment in the form of: Radiotherapy in 14 patients (29.2%) and Bisphosphonates
in 22 patients (45.8%). 8 patients (13.4%) were referred
for stem cell transplant, of which 5 patients (8.4%) had successful stem
cell transplant done and are still under regular follow-up.During this 6
year period, 24 out of 60 patients diagnosed with Multiple Meyloma
have died (40%). Survival range for these patients was between 1 month
and 45 months (Mean survival of 37.5 months). 60% of the patients
diagnosed are still under regular follow-up. Conclusions. Our retrospective
study of Myeloma patients in our hospital showed that a significant
percentage of patients (20%) had stable Myeloma with no organ damage
and did not require treatment. Usual presenting symptoms were less
pronounced than in other studies, reflecting possibly early diagnosis.
Only 13.8% of patients were referred for assessment for autologous
stem cell transplant, however only 8.4% had successful stem cell transplant.
0679
MYELOMA-EPIDEMIOLOGY: FARMERS ARE AT HIGHER RISK OF MYELOMA IN THE UK
D. Hofer, 1 S Zhang, 2 C Chapman1 1 University Hospitals of Leicester, LEICESTER; 2 MRC Toxicology Unit,(Bioinformatics),
LEICESTER, United Kingdom
Background. There are several publications which have shown an epidemiological
relationship between plasma cell dyscrasias or myeloma
and farming in general 1,2 or only some specific kinds of farming (e.g. cultivating
potatoes or sheep farming) in several different countries like the
U.S., France or Norway and Sweden. 3,4,5, 6,7 There are however no data so
far published for the UK. Aims. We wanted to demonstrate for the U.K.
that farmers are more prone to develop myeloma than other occupational
groups as has been shown for other countries. Methods. A survey was
conducted amongst the patients with myeloma or MGUS in the years
2005-2006 regarding their occupation and patients were grouped into
patients from city areas versus county areas according to their postcode.
The Leicester Royal Infirmary Haematology Department is the single
referral centre for the whole of Leicester city and rural Leicestershire
ensuring that both patient groups are represented without any geographical
selection bias. Results. A total of 255 patients with myeloma or
MGUS were surveyed, out of these 8 were farmers. Assuming a total
haematologica/the hematology journal | 2007; 92(s1) | 253