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

Anemia and Bone marrow failure
0061
GRANULOCYTE TRANSFUSION IN THE MANAGEMENT OF PATIENTS WITH
HEMATOLOGICAL MALIGNANCIES AND SEVERE INFECTION
H. Cherif, 1 U. Axdorph, 2 M. Kalin, 2 M. Björkholm2 1 Karolinska University Hospital, STOCKHOLM; 2 Karolinska University Hospital
and Ins., STOCKHOLM, Sweden
Background. Infection associated with chemotherapy induced neutropenia
continues to be a major cause of morbidity and mortality in
patients treated for hematological malignancies. The role of granulocyte
transfusion (GT) in this clinical setting has not been established. Aims and
Methods. We retrospectively analysed clinical characteristics and outcome
in a cohort of patients with hematological malignancies receiving
GT during neutropenia and severe infection. Results. A total of 30 patients
with a median age of 46 years (range 3-82) who had received one or more
GT, achieved from community donors after stimulation with G-CSF and
corticosteroids, were included. Acute leukemia (80%) and non-Hodgkin
lymphoma (17%) predominated as underlying hematological malignancy.
All patients had severe (absolute neutrophil count 5 years). Mortalities at 30-days and
6-months post GT were 40% and 72%, respectively. GT was well tolerated
and only 2 patients needed antihistamines/corticosteroids to avoid
reactions. Conclusions. A substantial proportion of severely ill patients
with complicated febrile neutropenia benefited from GT. Despite high
mortality, the presence of long time survivors motivates further evaluation
using well designed randomised prospective trials to evaluate the
efficacy of this intervention in patients with hematological malignancies.
0062
ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION FROM MATCHED RELATED
DONORS IN FANCONI ANEMIA PATIENTS: THE TUNISIAN EXPERIENCE WITH TWO CONDI-
TIONING REGIMENS
L. Torjemane
Centre National de Greffe de Moelle Osse, TUNIS, Tunisia
We transplanted twenty-four patients with Fanconi anemia (FA) using
matched related donors (MRD) between January 1999 and June 2006.
The patients were assigned to the following groups according to the
conditioning regimen: group 1 (N=17), low-dose cyclophosphamide (CY;
40 mg/kg)-busulfan (BU; 6 mg/kg) conditioning regimen; group 2 (N=7),
low-dose CY- Fludarabine (120 mg/m2 ) conditioning regimen. Median
age at transplantation was 10 years (range:3-22 years). Graft-versus-host
disease (GVHD) prophylaxis included cyclosporine A and methotrexate
(MTX; 5mg/m2 at day 1, 3, 6). Twenty-three patients engrafted (for an
absolute neutrophil count >0.5×109 /L) after a median time of 12 days
(range,10-16 days). Eighteen patients (78%) had sustained grafts, whereas
five others of the group 1 (29%) rejected grafts between day +39 and
22 months after transplantation. Four of them are still alive after successful
second PBSC transplantation and one died. All of the group 2 patients
had sustained grafts without major toxicity. Acute and chronic GVHD
occurred, respectively, in 17% and 9% of only group1 patients. After a
median follow-up of 53 months (range, 9-93 months), nineteen patients
are alive. The Kaplan-Meyer survival is 79% at 7 years. We conclude
that, in FA patients, fludarabine-based conditionning allowed engraftment
with low toxicity.
0063
ALEMTUZUMAB PLUS CYCLOSPORIN A FOR TREATMENT OF PATIENTS WITH BONE
MARROW FAILURE SYNDROME
H. Kim, J.-H. Park, S.-J. Shin, Y.J. Min
Ulsan University Hospital, ULSAN, South-Korea
Patients with bone marrow failure syndrome are suffered from
cytopenia and resulting requirement of transfusion. Allogeneic
hematopoietic stem cell transplantation (alloHSCT) is the standard therapy
for severe aplastic anemia (AA) with suitable sibling donor. However,
it is true in many cases that alloHSCT can not be performed even
in very severe AA due to no suitable donor. In that regards, immunosuppressive
therapy (IST) is another option for those patients. Alemtuzumab
(ALM) targets not only B-cell but also Tcell, which is known to play
an important role in pathogenesis of bone marrow failure syndrome
(BMFS). Although there were several reports of ALM for immune
cytopenia and pure red cell aplasia, no report were published regarding
ALM for BMFS. We studied the feasibility of ALM and cyclosporine A
(CsA) for patients with BMFS. Inclusion criteria were transfusiondependent
BMFS including AA, MDS, PNH. Prior IST or other drugs for
BMFS except for ALM were not excluded. ALM was infused for 3 days
(10 mg on day 1, 20 mg on day 2, and 30 mg on day 3) along with CsA
for at least 6 months. Prophylactic ciprofloxacin, acyclovir, and bactrim
were given for first 2 months. Without partial response (PR) or more
within 6 months, subsequent alloHSCT or antithymoglobulin therapy
was allowed. Total 12 patients were enrolled. Female were 9 (75%).
Median age was 43 (16-74) years. Disease were 9 severe AA, 2 moderate
AA and 1 hypoplastic MDS. All patients were transfusion-dependent.
Median amount of transfused red cell, platelet concentrate and
platelet apheresis prior to ALM-CsA were 2, 2 and 7 units, respectively.
Prior therapy were none in 9 (75%), anti-thymoglobulin in 2 (16.7%),
and danazol in 1 (8.3%). Only 1 patient could not receive full scheduled
CsA because the patient underwent alloHSCT. Median follow-up was
326 (140-466) days. Response to ALM-CsA were none response in 7
(58.3%), PR in 4 (33.3%), and CR in 1 (8.3%). Therefore, overall response
rate was 4/12 (41.7%). Median time to response was 47 (24-71) days (Figure
1). PR was converted to CR in 1 patient at 12 months after treatment.
Neither fever nor skin rash during treatment did not affect the possibility
of response to ALM-CsA (p=0.523, p=0.523, respectively). Toxicity
during ALM was fever in 3 (25%), skin rash in 3 (25%), G1 ALT elevation
in 7, G1 AST elevation in 4, and hyperbilirubinemia in 3 (G1 in 2,
G3 in 1). There were no anaphylaxis, serum sickness or CMV reactivation.
In spite of small number, AML-CsA showed comparable response
rate within 3 months after treatment. Therefore in conclusion, ALM-CsA
is feasible and tolerable therapy for BMFS.
Figure 1. Time to response.
12 th Congress of the European Hematology Association
haematologica/the hematology journal | 2007; 92(s1) | 23