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INTRODUCTION: The use of Peripherally Inserted Central Catheters (PICCs) has increased significantly in the last years due to their advantages compared to the other types of central catheters: easier and protocolized insertion by specialized nurse-led teams; cost-effectiveness; ease of management, ... However, an increase in the incidence of Catheter Related Thrombosis (CRT) has been observed with this type of device, especially in cancer patients and in the critical care setting. The main objective of this study is to determine the incidence of PICC related deep venous thrombosis in oncologic and onco-hematologic patients at the Donostia University Hospital, in Spain. The secondary outcome is the identification of possible risk factors associated with this event. METHODS: Using the database created and handled by the nurse-led Intravenous Therapy Team (ITT) in our center, in which all inserted PICCs are prospectively and consecutively included since 2011, a retrospective analysis was conducted on oncology and hemato-oncology-derived adult patients (over 18 years old) with a PICC inserted between May 15th 2018 and December 15th 2019. In the total population, several characteristics of the patient, of the PICC and of the thrombotic event were descriptively analyzed, and a bivariant analysis of four potential risk factors was carried out using Pearson's Chi-squared test. Patient and CRT treatment-associated risk factors were more exhaustively analyzed in the subgroup of patients with CRT. The missing data were obtained from the electronic clinical history records. RESULTS: The final study sample consisted of 1024 PICCs (n=1024), 19,10% (n=313) derived from the Hematology department and 43,62% (n=715) from the Oncology department (tables 1 and 2). The global incidence of CRT was 4,9% (n=50): 5.8% in hematologic patients and 4.5% in patients derived from Oncology. In the bivariant analysis no significant association was found between the selected potential risk factors (department of origin, PICC lumen number, PICC material and the catheter-to-vessel ratio) and CRT (table 3). In terms of the treatment administered to patients presenting CRT, in 80% of the cases (n=40) a Low Molecular Weight Heparin (LMWH) at therapeutic dose was initiated; in 10% (n=5) LMWH at a lower dose, and in 2 patients treatment could not be initiated because of thrombopenia. Finally, the PICC was withdrawn in only 8 patients after the diagnosis of the thrombotic event. CONCLUSIONS: The majority of the studies on PICC associated venous thrombosis in cancer patients are small, observational, retrospective, and without comparison groups. Here we present a work with an important sample size, a homogeneous population and with a prospective data collection. The CRT incidence has been similar to that described in the literature and significant association has not been found between the included potential risk factors and CRT. In conclusion, this study reflects the need of more trials on this subject, in particular to identify CRT risk factors in order to design effective prevention strategies. Disclosures No relevant conflicts of interest to declare.

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To determine the incidence and outcome of hepatic veno-occlusive disease (VOD) after blood or marrow transplantation (BMT), we prospectively evaluated all consecutive patients receiving a BMT during a 6-month period in participating EBMT centers. All of them were evaluated for occurrence of VOD according to previously defined clinical criteria. The clinical course, outcome, value of prophylactic and therapeutic interventions, and the influence of previously described risk factors were analyzed. During the study period, 1,652 BMT were performed in 73 centers. VOD was diagnosed in 87 patients (5.3%; 95% confidence interval [CI], 4.2% to 6.4%). Fifty-six of 631 allogeneic BMT (8.9%) and 31 of 1,010 autologous BMT (3.1%) developed this complication (P.0001). VOD was classified as mild in 7 (8%), moderate in 56 (64.4%), and severe in 24 (27.6%) cases. Sixteen patients died of VOD (corresponding to 1% of the whole series, 18.4% of VOD patients, and 66.7% of severe VOD). The use of unfractionated heparin did not significantly decrease the incidence of VOD. Independent variables associated with an increased risk of VOD were allogeneic BMT (relative risk [RR], 2.8; P.001), pre-BMT elevation of serum aspartate aminotransferase (RR, 2.4; P =.001), high-dose cytoreductive therapy (RR, 2.3; P =.003), Karnofsky performance score less than 90% (RR, 2.7; P =.006), and prior abdominal radiation (RR, 2.9; P =.03). In conclusion, this prospective study shows that (1) the incidence of VOD is lower than that reported in smaller studies from single centers, (2) about one fourth of cases of VOD progress to severe disease, (3) main risk factors have a major impact on incidence of VOD, and (4) the use of prophylactic unfractionated heparin does not seem to reduce the incidence of VOD.

Abstract 3099 Background: Despite the fact that allogeneic SCT currently offers patients with high risk AML the best chance of cure, we've aimed to investigate the outcome of AML patients who have undergone ASCT in our center, considered as one of spanish reference hospital in SCT, and the parameters that have been able to influence in relapse rate (RR), overall survival (OS) and relapse free survival (RFS). Methods: Retrospective study in 121 AML patients who have undergone ASCT between 1988 and 2010. The analysis has been performed in 95 patients (50 male and 45 female) by excluding 26 acute promyelocitic leukemias (APL): 62 patients until 1999 and 33 since 2000. Median age was 45 [18–74] and median lecocyte count, 17250/μL [1000–318000]. 90% were “de novo” AML and 92% were in first complete remission (1°CR). Cytogenetic risk was as follows: 64% intermediate, 29% high and 7% low. Conditioning regimens were oral BuCy (62%), CyTBI (24%) and intravenous BuCy (12%). Stem cell source was bone marrow (BM) in 46 patients (48%), but the use of peripheral blood (PB) was generalized since 1997. Colony-stimulating factors were used in 52% of total patients. Median time from last treatment was 94 days [30–285]. Results: The median follow-up of this study was 125 months [0–216]. OS at 1, 3 and 5 years were 59%, 46% and 44%. RFS at 1, 3 and 5 years were 60%, 49% and 48%. There was no relapse after 5 years from ASCT. Early mortality (before day +100) was 12% (9 in 11 patients between 1988 and 1999) and late mortality was 47% (34 in 45 patients because of relapse, with no significant difference between 1988–1999 and 2000–2010). Secondary malignancies incidence was 13% (5 in 6 patients suffered from haematologic neoplasms: 4 MDS at 43, 89, 90 and 97 months and 1 Follicular Lymphoma at 50 months), median age of these patients was slightly higher (53, 5 years old) and none of them had received TBI. Multivariable analysis showed that RFS at 5 years was influenced by: disease status at ASCT (55% if 1°CR vs 0% if ≥2°CR/PR/refractory disease, p Conclusions: ASCT is an effective procedure of cure in AML patients (global RFS of 48% at 5 years), even in high cytogenetic risk (38% with no significant difference), offering its best outcomes in young patients diagnosed of “de novo” AML without hyperleucocytosis, who have undergone the transplantation in 1°CR since 2000. Disclosures: No relevant conflicts of interest to declare.

Abstract 4498 Background: Giving the fact that allo- SCT currently offers patients with high risk AML the best chance of cure, we`ve aimed to investigate the outcome of AML patients who have undergone allo-SCT in our center, considered as one of spanish reference hospital in SCT, and the parameters that have been able to influence in relapse rate (RR), overall survival (OS) and relapse free survival (RFS). Methods: Retrospective study in 192 AML patients who have undergone allo-SCT between 1982 and 2010. The analysis has been performed in 171 patients (85 male and 86 female) by excluding 21 acute promyelocitic leukemias (APL): 65 patients until 1999 and 106 since 2000. Median age was 37 1874 and median lecocyte count, \batchmode \documentclass[fleqn,10pt,legalpaper]{article} \usepackage{amssymb} \usepackage{amsfonts} \usepackage{amsmath} \pagestyle{empty} \begin{document} \(13400/\hbox{ L }\frac{470}{250000}\) \end{document}. 82 were de novo AML and 87 were in morfologic complete remission (70 in first CR). 14 patients had received a previous SCT. Cytogenetic risk was as follows: 55 intermediate, 34 high and 11 low. Conditioning regimen was ablative in 162 patients: CyTBI (36), BuCy (31), BuFlu (30) and others (3). 130 patients (76) underwent a related allo-SCT (95 of them were matched) and 41 patients (24), an unrelated allo-SCT (64 of them were matched). Stem cell source was bone marrow (BM) in 146 patients (85) and only 3 patients received umbilical cord (UC). Graft versus host disease (GvHD) prophilaxis was based on Ciclosporine in 150 patients (88). Median time from last treatment was 73 days 12268. Results: The median follow-up of this study was 61 months 1317. OS at 1, 3 and 5 years were 57, 44 and 40. RFS at 1, 3 and 5 years were 62, 50 and 45. Early mortality (before day 100) was 26 (43 until 1999 and 15 since 2000, p0,0001): 18 patients because of infections, 10 because of toxicity, 9 because of disease and 7 because of EICH. Late mortality was 27 (more than the half because of relapse, with no significant difference between 19881999 and 20002010). Cumulative relapse incidence at 5 years was 35, with a median time of relapse of 4 months. Secondary malignancies incidence was 5. Multivariable analysis showed that Transplantation Related Mortality (TRM) was influenced by: year of allo-SCT (OS at 5 years of 49 if 20002010 vs 28 if 19821999, p0,0001), late engraftment (p0,002) and severe acute GvHD (OS at 5 years of 45 if no evidence/grade I-II vs 25 if grade III-IV, p0,071). The other important parameters which lost its univariable analysis significance were donor type, recipient age and conditioning regimen. No difference was found in case of HLA and ABO discordance or donor/recipient CMV status. Multivariable analysis also showed that RR and RFS at 5 years was influenced by: disease status at allo-SCT (50 if 1CR vs 0 if 2CR/PR/refractory disease, p0,002), chronic GvHD (67 if present vs 41 if absent, p0,035) and leucocyte count at diagnosis (54 if 20000/ L vs 37 if 20000/ L, p0,038). The other important parameters which lost its univariable analysis significance were cytogenetic risk, initial induction response and positive minimal residual disease (MRD) before allo-SCT. No diference was found in case of ethiologic classification or stem cell source. Conclusions: Allo-SCT is a curative procedure in AML patients (global RFS of 50 at 3 years), specially when disease is under control and patient develops chronic GvHD. In the last decade, there have been important improvements in the procedure which have led to a significant decrease in TRM, and consequently, a significant increase in OS. Disclosures: No relevant conflicts of interest to declare.

Abstract 3077 Blastic plasmacytoid dendritic cell neoplasm (BPDC), formerly known as blastic NK cell lymphoma, is a rare hematopoietic malignancy preferentially involving the skin, bone marrow and lymph nodes. The overall prognosis of BPDC is dismal. Most patients show an initial response to acute leukemia-like chemotherapy, but relapses with subsequent drug resistance occur in virtually all patients resulting in a median overall survival of only 9–13 months. However, anecdotal long-term remissions have been reported in young patients who received early myeloablative allogeneic stem cell transplantation (alloSCT). We therefore performed a retrospective analysis of patients identified in the EBMT registry in order to evaluate the outcome of autologous stem cell transplantation (autoSCT) or alloSCT for BPDC. Eligible were all patients who had been registered with a diagnosis of BPDC or Blastic NK cell lymphoma and had received autologous stem cell transplantation (autoSCT) or alloSCT in 2000–2009. Centres were contacted to provide a written histopathology and immunophenotyping report and information about treatment and follow-up details. Patients who did not have a diagnostic score ≥ 2 as proposed by Garnache-Ottou et al. (BJH 2009) were excluded. RESULTS: Overall, 139 patients could be identified in the database who fulfilled the inclusion criteria (alloSCT 100, autoSCT 39). Of 74 patients for whom the requested additional information could be obtained, central review confirmed the diagnosis of BPDC in 39 patients (34 alloSCT, 5 autoSCT). The 34 patients who had undergone alloSCT had a median age of 41 years (range: 10–70 years), were transplanted from a related (n=11) or unrelated donor (n=23); received peripheral blood stem cells (n=9), bone marrow stem cells (n=19) or cord blood (n=6); and had been treated with a reduced intensity conditioning regimen (RIC, n=9) or myeloablative conditioning (MAC, n=25). Nineteen of 34 patients were transplanted in CR1. After a median follow up time of 28 months (range: 4–77+ months), 11 patients relapsed (median time to relapse: 8 months, range: 2–27 months) of whom 8 died due to disease progression. 9 patients died in the absence of relapse. No relapse occurred later than 27 months after transplant. Median disease free survival (DFS) was 15 months (range: 4–77+ months) and median overall survival (OS) was 22 months (range: 8–77+ months; Figure 1a). However, long-term remissions of up to 77 months after alloSCT could be observed. Patients allografted in CR1 tended to have a superior DFS (p=0.119) and OS (p=0.057; Figure 1b). MAC was associated with a better OS (p=0.001) which was attributable to the significantly higher non-relapse mortality (NRM) rate of patients after RIC (p=0.014), who had been significantly older (age RIC: 56 years, age MAC: 36 years, p=0.0014). The relapse rate was not different in patients after RIC and MAC, respectively. However, there was no survivor after RIC. Median age in the autoSCT group was 47 years (range: 14–62 years). Three of 5 patients were transplanted in CR1 of whom 1 patient relapsed after 8 months, 1 patient experienced treatment related mortality and 1 patient remained in CR for 28 months. The 2 remaining patients had more advanced disease at autoSCT and relapsed 4 and 8 months thereafter. CONCLUSION: AlloSCT is effective in BPDC and might provide curative potential in this otherwise incurable disease, especially when performed in CR1. However, it remains to be shown by prospective studies if the potential benefit of alloSCT in BPDC is largely due to conditioning intensity, or if there is a relevant contribution of graft-versus-leukemia activity. Disclosures: Tilly: Seattle Genetics, Inc.: Membership on an entity's Board of Directors or advisory committees, Research Funding; Celgene: Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau, Travel/accommodations/meeting expenses; Genentech: Membership on an entity's Board of Directors or advisory committees; Roche: Membership on an entity's Board of Directors or advisory committees, Research Funding; Amgen: Research Funding, Speakers Bureau; Pfizer: Speakers Bureau; Janssen Cilag: Speakers Bureau.

Both autologous stem cell transplantation (autoSCT) and reduced intensity allogeneic stem cell transplantation (RIST) may be considered as treatment options in patients with relapsed follicular lymphoma (FL). It is currently unknown which transplant strategy is optimal in patients with FL. We have therefore conducted a retrospective comparison of patients who have undergone an autoSCT or a RIST. Adult patients undergoing a 1st transplant procedure and reported to the EBMT between Jan 1998 and Dec 2005 were included. 1394 patients underwent an autoSCT and 110 a RIST with median ages of 51 (20–73) and 50 (32–65) respectively. Patients undergoing a RIST had significantly more advanced disease (stage IV 74% vs 60% p=0.01) and B symptoms (39% vs 27% p=0.05) at diagnosis but there was no difference in the size of the largest mass or the LDH. The median time from diagnosis to transplant was 27 months for the whole group but was significantly longer for patients undergoing a RIST (34 months vs 26 months p=0.005). Patients undergoing a RIST had received more lines of prior therapy (61% 3 or more lines vs 34% for those undergoing autoSCT p

Background: fludarabine (F) is licensed for the management of indolent non Hodgkin’s lymphoma in countries such as Canada and Switzerland. Clinical evidence suggests that fludarabine monotherapy is as least as effective, than conventional therapies such as cyclophosphamide, vincristine, prednisone (CVP) for the first and second line treatment of NHL achieving objective response rates. The non Hodgkin’s lymphomas are a heterogeneous group of lymphomas involving predominantly malignant populations of B lymphocytes, although T or natural killer type (NK) may be present. Aims: to assess the efficacy, safety and quality of life of F in previously untreated indolent NHL in a Group of Medical Institutions in Uruguay during 11 years (1995–2006). Methods: 98 patients between the period 1995 – 2006 were evaluated. 72 of them received the intravenous formulation (1995–2006) and 26 the oral one (2002–2006). Age: 38 – 85 years old, media 58 years old. Gender: male 52% & female 48%. Inclusion criteria for NHL-LG was: non previous treatment, Ann Arbor stage III or IV (nodal or extradonal), a meassurable mass, age > 18 years old, WHO performance status 0–2 and written informed consent. WHO performance status 0: 60 pts, 1: 24 pts and 2: 14 patients. Ann Arbor staging: IIIA:16/98 IIIB:9/98 IVA:51/98 and 22 IVB. Treatment: all the patients received (minimum): 6 cycles of i.v. Fludarabine (Fludara®, Schering) 25 mg/m2/daily (5 days) e/30 days or Oral Fludarabine, 40 mg/m2/daily (5 days), 6 cycles. Results: on this NHL-LG cohort the overall response rate (PR+CR) was 78% (ORR), 44% complete response and 34% partial response. Stable disease 2%. Progressive disease 23%. Time to progression 15 months. Overall survival at 60 months was 68%. LDH in serum was an adverse prognostic factor for time to progression and overall survival. Safety: on the 622 cycles in 98 patients, the toxicity was: 1 AIHA, 2 pancytopenia, 1 plaquetopenia. Grade 3–4 infection rate was 1,3%. No alopecia was observed in any patient. Mortality rate: 1,02% (1/98 patients). Other adverse factors to overall survival were, age over 65 years old (p=0,0001) and hepatic impairment (p=0,0001). Toxicity: (WHO>2): granulocytopenia 18%, thrombocytopenia 10%, infection 6%. Although fludarabine-treated patients experienced more significant myelossuppresion, no difference in the treatment group was demonstrated. Causes of death: sepsis 10%, associated disease 46%, second malignancy 7% and others 37%. Comparing oral with intravenous formulation in overall survival the results were: NHL-LG 77% vs 73% (p= NS). Conclusions: fludarabine monofosfate (Fludara®) is an effective and safe treatment for NHL-LG. The oral and intravenous formulations have a similar response rate in elderly and young patients. A longer follow up and a larger trial, might be needed to confirm these results in a multicenter, randomized study.

Background: fludarabine (F) has become the standard first line therapy for chronic lymphocytic leukemia (CLL) in younger patients. Treatment of early stage patients with chlorambucil without risk stratification has not been shown to prolong survival. In recent years effective and potentially curative approaches such as nucleosides analogues, stem cell transplantation or monoclonal antibodies have been developed. The attraction of monoclonal antibodies is based on selective targeting of tumor - relevant surface markers and a distinct mechanism of action (antibody-dependent cellular cytotoxicity). Aims: to assess the efficacy, safety and quality of life of F in previously untreated B-cell CLL patients in a group of medical institutions in Uruguay during 11 years (1995–2006). Methods: 168 patients between the period 1995 – 2006 were evaluated.120 of them received F intravenous formulation (1995–2006) and 48 the oral one (2002–2006). Age: 48 – 85 years old, media 67 years old. Gender: male 90, female 78. Inclusion criteria for B-cell CLL was Binet stages B, C and A progressive (Ap), 18 to 85 years old, non multiorganic failure, performance status 0 – 2 (WHO), written informed consent. First condition was non previous treatment. Staging: Binet A 12/168, B 116/168 & C 40/168. Treatment: as first line therapy all the patients received (minimum): 6 cycles of i.v. Fludarabine (Fludara®, Schering) 25 mg/m2/daily (5 days) e/30 days or Oral Fludarabine, 40 mg/m2/daily (5 days), 6 cycles. Results: on this B-cell CLL cohort the overall response rate (ORR) was 78% (CR+PR), 80% of them have immunophenotypic response. Safety: on the 1100 cycles in 168 patients, the toxicity was: 1 AIHA, 2 pancytopenia, 3 plaquetopenia. Grade 3–4 infection rate was 1,3%. No alopecia was observed in any patient. Kaposi sarcoma (0,7%). Mortality rate: 1,7% (3/168 patients). Other adverse factors to overall survival were, age over 65 (p=0,0001) and hepatic impairment (p=0,0001). Toxicity: (WHO>2): granulocytopenia 28%, thrombocytopenia 8%, infection 2%. Although fludarabine-treated patients experienced more significant myelosuppression, no difference in the treatment group was demonstrated. Causes of death: Richter 12%, sepsis 5%, associated disease 34%, second malignancy 17% and others 30%. Comparing oral with intravenous formulation in overall survival the results were: CLL 34% vs 36% (p= NS). Conclusions: fludarabine monofosfate (Fludara®) looks like an effective and safe treatment for B-cell CLL. The oral and intravenous formulations have a similar response rate in elderly and young patients. The challenge remains to integrate new information to apply novel therapies in a disease-specific and risk-adapted maner. A longer follow up and a larger trial, might be needed to confirm these results.

Expression of CD38 and /or ZAP 70 and chromosome abnormalities such as deletions of 11q23 and 17p13 have been previously identified by several groups as predictors of disease progression and decrease overall survival in Chronic Lymphocytic Leukemia (CLL). Moreover, few are described about the relationship between ZAP70/CD38 status and their genomic aberrations associated. We analysed 86 patients (median age: 70 yrs; female/male: 28/58) diagnosed of CLL between 1980 and 2005. Cytoplasmic ZAP-70 expression and cell surface expression of CD38, was determined by flow cytometry in CD19+ CD5+ B-CLL cells. Trisomy 12 and deletions in 13q14, 11q22–23 and 17p13, were detected by the interphase cytogenetic fluorescence in situ hybridization (FISH). Biological study in patients diagnosed before 2001 was done on cryopreserved blood cells. Concordant expression of ZAP-70 and CD38 was seen in 66 of 86 patients (76%). Fifty-one patients were ZAP-70- and CD38-, and 15 patients were ZAP-70+ and CD38+. Discordant ZAP70/CD38 status was present in 20 patients. Cytogenetic study identified chromosome aberrations in 85% of patients. Deletions in band 13q14 (43%), followed by trisomy 12 (21%), deletions in 11q22–23 (11%) and in 17p13 (10%) were the most frequent abnormalities. Normal karyotype was presented in 28% of patients and complex karyotype in 11%. In the concordant ZAP70-CD38- cases and ZAP70+ CD38+, 13q14 aberration (47, 9%) and normal karyotype (42%) respectively, were more frequent than in the other groups but in our study this differences were not statistically significant. Moreover deletion in 11q22–23 (25%) was seen statistically significant, p = 0, 05, in patients with concordant expression of ZAP70+ and CD38+. Patients with discordant status in the expression of ZAP70 and CD38, were statistically associated with trisomy 12 (38%) p = 0,002. In conclusion, in our study we find that expression in ZAP70 and CD38 is associated with a different genomic aberration. Deletion in 13q14 appears more frequent in patients with concordant ZAP70- and CD38- expression. Trisomy 12 is associated with discordant status expression. Finally, the worse prognostic group ZAP70+ and CD38+ is associated with a normal karyotype or deletions in 11q22–23. Expression of ZAP 70 and CD38 and genomic aberrations ZAP70+ CD38+ ZAP70- CD38- Discordant status p-value Del 11q22-23 25% 4,2% 12,5% 0,05 Del 17p13 8,3% 4,2% ---- 0,52 Trisomy12 8,3% 10,4% 37,5% 0,002 Del 13q14 16,7% 47,9% 31,3% 0,10 Normal 41,7% 33,3% 18,8% 0,39 Complex Karyotype 20% 5,9% 20% 0,13

Chronic Lymphocytic Leukemia (CLL) is heterogeneous and there are two clinical forms with different outcome, in which initial stage and parameters of tumor burden, expression of CD38 and /or ZAP 70, chromosome abnormalities such as 11q23 and 17p13 are associated with a more aggressive course of disease. The aim of this study is to analyse clinical and biological characteristics and outcome of patients with complex karyotype, and to asses if there are differences with other genomic aberrations. Fifty patients diagnosed of CLL between 1980 and 1994 were studied (median age: 71 yrs; female/male: 18/32; median follow up 6.6 yrs.). Biological studies with flow cytometry and fluorescence in situ hybridization were done on cryopreserved blood cells. Clinical stages at diagnosis were: Rai stage 0: 56%; I: 20%; II: 16%; III: 6%; IV: 2%. Binet A: 72%; B: 20%; C: 8%. At the time of diagnosis 37% of patients needed treatment and 61% presented disease progression. At the time of this study, 10% of patients are alive with a median follow up of 15.3 yrs. Seven patients (28%) presented complex karyotype. At the time of diagnosis 43% patients needed treatment and clinical stages were: Rai stage O: 29%, I: 14%, II: 43%, III: 14%, IV: 0%. Binet A: 43%; B: 43%; C: 14%. Median overall median survival was 72 months. The most frequent genomic aberrations associated with complex karyotype were: del (17p13) and trisomy 12 (60 %). Cellular expression of CD38 and ZAP 70 was not able to separate this group. Compared with other chromosome abnormalities, there were not important differences in clinical stage at diagnosis or expression of CD38 or ZAP 70, but overall median survival could distinguish three groups: del 11q22-23 or del 17p13 (34 months, p < 0,05) complex karyotype (72 months), and trisomy 12 or del 13q14 (79 months, p > 0,05). In conclusion, complex karyotype is frequently formed by del 17p13 and trisomy 12 and identifies a subgroup of patients with better prognosis compared with isolated p53 deletion. More studies have to be realized to determine if trisomy 12 is a cell defence mechanism.

Allogeneic bone marrow transplantation (BMT) was carried out on a 5- year-old boy with congenital hypoplastic anemia (CHA), who did not respond to corticosteroids and who was displaying signs of progressive hemosiderosis. Pretransplant preparation had to be modified because respiratory failure and cerebral edema supervened. This preparatory regimen consisted of busulfan (2 mg/kg for four days), cyclophosphamide (50 mg/kg for one day), and total body irradiation (750 rad). Hemopoiesis was completely restored and is still maintained 650 days after transplantation. This is the second published report on the use of BMT to treat a patient with CHA, and it is the first time it has resulted in long-term survival. BMT should be considered for patients with CHA who do not respond to corticosteroids.