Gérard Socié

Paris Diderot University, Lutetia Parisorum, Île-de-France, France

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Publications (550)3355.44 Total impact

  • Leukemia. 08/2014;
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    ABSTRACT: We analyzed the outcome of 537 adolescents (age 12-18 years) with idiopathic aplastic anemia included in the data-base of the Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation comparing (i) matched family donor hematopoietic stem cell transplantation performed as first line treatment with (ii) front-line immunosuppressive therapy not followed by subsequent transplant given for failure and with (iii) Hematopoietic Stem Cell Transplantation performed after failed front-line immunosuppressive therapy . Overall Survival was 86% in matched family donor hematopoietic stem cell transplantation group , 90% in patients given front-line immunosuppressive alone (those who did not fail this treatment and who did not receive subsequent rescue hematopoietic stem cell transplantation) and 78% in subjects who underwent hematopoietic stem cell transplantation post failed font-line immunosuppressive therapy (p=0.14). Event Free Survival in the same groups was respectively 83%, 64% and 71% (p= 0.04). Cumulative incidence of rejection was 8% in in matched family donor hematopoietic stem cell transplantation and 9% in transplants post failed front- line immunosuppression (p=0.62). Cumulative incidence of acute graft versus host disease was 12% in matched family donor transplants and 18% in transplants post failed immunosuppression (p=0.18). Chronic graft versus host disease was higher in matched family donor hematopoietic stem cell transplantation (8%) than in transplants post failed immunosuppressive therapy (20%) (p=0.0009). Cumulative incidence of post-therapy malignancies was 0.7% in matched family donor transplantations, 7% in transplantations post-failed immunosuppression and 21% after front-line immunosuppression (p= 0.0017). In the whole cohort in multivariate analysis, the diagnosis to treatment interval of ≤2 months positively affected overall survival whereas upfront immunosuppression alone ( with no subsequent rescue transplants) negatively affected event free survival. In transplanted patients interval diagnosis-treatment ≤2 months, first line matched family donor transplants provided a significant advantage in overall and event free survival. Aplastic anemia in adolescents has a very good outcome. If a matched family donor is available, hematopoietic stem cell transplantation using bone marrow cells is the first choice treatment. If such a donor is not available, immunosuppressive treatment may still be an acceptable second choice also because in case of failure, hematopoietic stem cell transplantation is a very good rescue option.
    Haematologica 08/2014; · 5.94 Impact Factor
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    ABSTRACT: Natural killer cells are the first lymphocyte subset to reconstitute, and play a major role in early immunity after allogeneic hematopoietic stem cell transplantation. Cells expressing the activating receptor NKG2C seem crucial in the resolution of cytomegalovirus episodes, even in the absence of T-cells. We prospectively investigated Natural Killer cells reconstitution in a cohort of 439 adult recipients who underwent non T-cell depleted allogeneic hematopoietic stem cell transplantation between 2005 and 2012. Freshly collected blood samples were analyzed 3, 6, 12 and 24 months after transplantation. Data were studied with respect to conditioning regimen, source of stem cells, underlying disease, occurrence of Graft-versus-Host Disease, and profiles of cytomegalovirus reactivation. In multivariate analysis, we show that the absolute numbers of CD56brightNatural killer cells at month 3 were significantly higher after myeloablative than after reduced intensity conditioning. Acute Graft-versus-Host Disease impaired reconstitution of total and CD56dimNatural killer cells at month 3. In contrast, high Natural killer cell count at month 3 was associated with a lower incidence of chronic Graft-versus-Host Disease, independently from a previous episode of acute Graft-versus-Host Disease and stem cell source. NKG2C+CD56dim and total Natural killer cell count at M3 was lower in patients reactivating CMV between month 0 and month 3, but expanded greatly afterwards. These cells were also less numerous in patients who experienced subsequent reactivation between month 3 and month 6. Our results advocate a direct role of NKG2C-expressing Natural killer cells in the early control of cytomegalovirus reactivation after allogeneic hematopoietic stem cell transplantation.
    Haematologica 08/2014; · 5.94 Impact Factor
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    ABSTRACT: Fanconi anemia is a rare and heterogeneous inherited disorder. The natural history of FA is characterized by marrow failure and a risk for clonal evolution. Evolution to acute myeloid leukemia is the main cause of premature mortality. Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) is considered the treatment of choice in this situation but the evolution is usually poor post-HSCT (due to treatment-related mortality). From August 2006 to December 2011, 6 consecutive patients with FA who received a sequential treatment with chemotherapy and reduced-intensity conditioning for clonal evolution in four different French institutions were reviewed. Five patients presented an AML and one a myelodysplastic syndrome. The sequential strategy consisted of a pre-transplant chemotherapy by fludarabine 30 mg/m2/d 5 days and cytarabine 1gr/m2x2/d 5 days with granulocyte-colony stimulating factor injections (FLAG) followed early after by a reduced-intensity conditioning [4 days cyclophosphamide 10 mg/kg , 4 days fludarabine 30 mg/m2, 2 days anti-thymocyte globulin (3.75 mg/kg) and TBI (2 Gy)]. The source of stem cells was cord blood for three patients (HLA 5/6 for 2 and 4/6 for one) and bone marrow of unrelated donors for others (HLA 9/1O for one and 10/10 for two). Graft versus host disease prophylaxis consisted in cyclosporine plus MMF. Median age of the patients at HSCT was 20.5 years (5-28). Pre-transplant chemotherapy and conditioning regimen were well tolerated. The median time between chemotherapy and the date of HSCT was 30 days (when neutrophil count reached values below 500/microL). All patients engrafted. Median time to engraftment was 26 days (14,21,25,27,31,35) for neutrophils and 29 days for platelets(21,25,29,29,35,42). Donor chimerism was complete at day 100 for 5 patients. Septicemias were encountered in three patients (Staphylococcus, Enterobacter, and Candida) at 1, 3 and 4 months after HSCT, respectively. One patient developed a diarrhea to Campylobacter jejuni one month after HSCT and another patient underwent meatotomy. Acute GvHD (1 grade I of the skin and 1 grade II in the gastrointestinal tract ) occurred in 2 patients . Both responded to steroid therapy. Chronic GvHD occurred in two patients (involve the skin). After a median follow-up (FU) of 30 months (5-72), all patients are still alive in complete remission from the clonal evolution. With a median FU of 30 months, all patients are alive and free from their original AML or MDS. The number of patients is little but the usual evolution is very poor post-HSCT in this particular situation. We thus believe this study supports the use of a sequential strategy (FLAG and RIC HSCT) in FA patients with AML or MDS.
    Haematologica 08/2014; · 5.94 Impact Factor
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    ABSTRACT: We examined risk of second solid cancers after allogeneic hematopoietic cell transplantation (AHCT) using reduced intensity/non-myeloablative conditioning (RIC/NMC). RIC/NMC recipients with leukemia/myelodysplastic syndrome (MDS) (n=2833) and lymphoma (n=1436) between 1995-2006 were included. In addition, RIC/NMC recipients 40-60 years of age (n=2138) were compared with patients of the same age receiving myeloablative conditioning (MAC, n=6428). The cumulative incidence of solid cancers was 3.35% at 10-years. There was no increase in overall cancer risk compared to the general population (standardized incidence ratio [SIR] 0.99, P=1.00 for leukemia/MDS and 0.92, P=0.75 for lymphoma). However, risks were significantly increased in leukemia/MDS patients for cancers of lip (SIR 14.28), tonsil (SIR 8.66), oropharynx (SIR 46.70), bone (SIR 23.53), soft tissue (SIR 12.92), and vulva (SIR 18.55) and skin melanoma (SIR 3.04). Lymphoma patients had significantly higher risks of oropharyngeal cancer (SIR 67.35) and skin melanoma (SIR 3.52). Among RIC/NMC recipients, age >50 years was the only independent risk factor for solid cancers (hazard ratio [HR] 3.02, P<0.001). Among patients age 40-60 years, when adjusted for other factors, there was no difference in cancer risks between RIC/NMC and MAC in leukemia/MDS patients (HR 0.98, P=0.905). In lymphoma patients, risks were lower after RIC/NMC (HR 0.51, P=0.047). In conclusion, the overall risks of second solid cancers in RIC/NMC recipients are similar to the general population, although there is an increased risk of cancer at some sites. Studies with longer follow-up are needed to realize the complete risks of solid cancers after RIC/NMC AHCT.
    Biology of blood and marrow transplantation: journal of the American Society for Blood and Marrow Transplantation 07/2014; · 3.15 Impact Factor
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    ABSTRACT: We performed a retrospective analysis on 421 adult patients who underwent unrelated cord blood transplantation (UCBT) for ALL. Median age was 32 years; 46% were in first CR (CR1), 32% in CR2 and 22% had advanced disease. Double UCBT was performed in 173 patients (41%). Myeloablative conditioning (MAC) was given to 314 patients (75%). Cumulative incidence (CI) of 60-day neutrophil recovery was 78%. CI of acute and chronic GVHD was 33 and 26%, respectively. Non-relapse mortality (NRM) at 2 years was 42%. Age⩾35 years (P<0.0001), advanced disease at UCBT (P<0.0001) and use of MAC (P<0.0001) were associated with increased NRM. Relapse incidence (RI) at 2 years was 28%; use of reduced intensity conditioning (RIC) (P=0.0002) was associated with increased RI. Two-year leukemia-free survival (LFS) was 39% for patients in CR1, 31% for CR2 and 8% for advanced disease. In multivariate analysis, factors associated with decreased LFS rate were: age ⩾35 years (P=0.034), use of MAC (P=0.032) and advanced disease (P<0.0001). These results show that UCBT is a valuable option to treat high-risk adult ALL when in remission. Strategies to decrease toxicity and relapse are needed to improve final outcomes.
    Bone marrow transplantation. 07/2014; 49(7):887-94.
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    ABSTRACT: This retrospective analysis compared two regimens of fludarabine combined with i.v. BU 6.4 mg/kg (FB2) or BU 12.8 mg/kg (FB4) for allografting of AML in first CR. A total of 437 patients (median age: 50 years) were administered FB2 (n=225, 51%) or FB4 (n=212, 49%). Median follow-up time was 28 months. Use of FB2 resulted in a longer time to neutrophil engraftment (17 vs 15 days, P<0.0001) but no difference in incidence of grade II-IV acute (P=0.54) or chronic GVHD (P=0.51). In patients <50 years of age, FB2 was associated with a higher 2-year cumulative incidence of relapse (33±6% vs 20±4%, P=0.04), but there was no difference in 2-year leukemia-free survival (LFS) (P=0.45), OS (P=0.53) or non-relapse mortality (P=0.17). In recipients ⩾50 years of age, FB2 resulted in better 2-year LFS (63±4% vs 42±7%, P=0.02) and OS (68±4% vs 45±7%, P=0.006); a lower 2-year non-relapse mortality, albeit not statistically significant (15±3% vs 29±6%, P=0.06), was observed with FB2. FB2 is an effective and well-tolerated regimen in patients ⩾50 years of age and does not compromise survival when used in patients <50 years undergoing allogeneic transplantation for AML in first CR.Bone Marrow Transplantation advance online publication, 30 June 2014; doi:10.1038/bmt.2014.133.
    Bone marrow transplantation. 06/2014;
  • Transplantation 06/2014; 97(12):e75-e77. · 3.78 Impact Factor
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    ABSTRACT: High-dose immunosuppressive therapy and autologous hematopoietic stem cell transplantation (HSCT) have shown efficacy in systemic sclerosis in phase 1 and small phase 2 trials.
    JAMA The Journal of the American Medical Association 06/2014; 311(24):2490-2498. · 29.98 Impact Factor
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    ABSTRACT: We have prospectively evaluated the safety and efficacy of a clofarabine, intravenous busulfan and antithymocyte globulin-based reduced-toxicity conditioning (CloB2A2) regimen before allogeneic stem cell transplantation. Thirty high-risk patients (median age: 59 years; acute myeloid leukemia n=11, acute lymphoblastic leukemia n=13; myelodysplastic syndrome n=5, biphenotypic leukemia n=1) were included in this phase 2 study. At time of transplant, 20 and 7 patients were in first and second complete remission, respectively, while 3 myelodysplastic syndrome cases were in response or have not been previously treated. The CloB2A2 regimen consisted of 30 mg/2/day clofarabine for 4 days, 3.2 mg/Kg/day busulfan for 2 days and 2.5 mg/kg/day antithymocyte globulin for 2 days. Median follow-up was 23 months. All patients engrafted. The one-year overall survival, leukemia-free survival, relapse incidence and non-relapse mortality were 63+-9%, 57+-9%, 40+-9%, and 3.3+/-3%, respectively. When comparing acute myeloid leukemia/myelodysplastic syndrome versus acute lymphoblastic leukemia/bi-phenotypic patients, the one-year overall and leukemia-free survivals were 75+/-10% vs 50+/-13%, p=0.07; and 69+/-12% vs 43+/-13%, p=0.08, while one-year relapse incidence was 25±11% vs 57+/-14%, p=0.05. The CloB2A2 regimen prior to allogeneic stem cell transplantation is feasible, allowing for full engraftment and low toxicity. Disease control appears to be satisfactorily, especially in acute myeloid leukemia/myelodysplastic syndrome. The trial has been registered at www.clinicaltrials.gov no. NCT00863148.
    Haematologica 06/2014; · 5.94 Impact Factor
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    ABSTRACT: Activating anti-angiotensin type 1 receptor antibodies (AT1R-AA) have been described in patients with systemic scleroderma, an auto-immune disorder with clinical fibrotic features. Chronic graft-versus-host disease (cGvHD) after hematopoietic stem cell transplantation may have clinical fibrotic features, whose pathogenesis may be similar with systemic sclerosis.
    Transplantation 05/2014; · 3.78 Impact Factor
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    ABSTRACT: Non-relapse mortality (NRM) after allogeneic hematopoietic stem cell transplantation (alloHSCT) can be predicted by the hematopoietic cell transplantation comorbidity index (HCT-CI) and the European Group for Blood and Marrow Transplantation (EBMT) score, which are composed of different parameters. We set out to integrate the parameters of both scores in patients with acute myeloid leukemia (AML) in first complete remission (CR1) receiving reduced intensity conditioning (RIC) alloHSCT. All parameters from the HCT-CI and the EBMT-score with the addition of patient and donor cytomegalovirus serology were evaluated in 812 patients by multivariable analysis with end-point NRM at 2 years. Subsequently, 16 parameters were selected based on hazard ratio >1.2, and were incorporated into a novel score, which was further internally validated by bootstrapping. Both the HCT-CI and the EBMT-score showed relatively weak predictive value, whereas the integrated score allowed to identify three clearly distinct risk groups with 2-year NRM estimates of 8±2% (low-risk), 17±2% (intermediate-risk) and 38±4% (high-risk), which also translated in prediction of overall survival. Collectively, integration of the most dominant parameters from the HCT-CI and the EBMT-score allowed to develop a simple and robust, integrated score with improved prediction of NRM for AML patients proceeding to RIC alloHSCT in CR1.Leukemia advance online publication, 10 June 2014; doi:10.1038/leu.2014.164.
    Leukemia. 05/2014;
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    ABSTRACT: Previous studies on regulatory T-cell (Treg) reconstitution after allogeneic hematopoietic SCT (HSCT) have suggested that, within the GVHD process, imbalance between effector T cells and Tregs may be more important than the absolute numbers of circulating Tregs. No study has analyzed naive vs memory Treg reconstitution in a longitudinal cohort with large numbers of patients. The reconstitution of total and subsets of Treg was prospectively analyzed by flow cytometry in 185 consecutive recipients at 3, 6, 12 and 24 months after allogeneic HSCT. The levels of total, naive and memory Tregs increased, mainly within the memory subset, but remained lower than healthy controls up to 2 years after transplantation. Reduced-intensity conditioning and peripheral blood (PBSC) as the source of stem cells were associated with better 3-month reconstitution. In multivariate analysis, PBSC, recipient age ⩽25 and no anti-thymoglobulin in the conditioning regimen were associated with a better Treg reconstitution. Naive Treg long-term reconstitution was mainly influenced by recipient age. Whereas prior acute GVHD impaired Treg reconstitution, Treg subsets (absolute numbers and frequencies relative to CD4(+) T-cell subsets) at 3, 6 and 12 months after HSCT were not associated with the occurrence of a later episode of chronic GVHD.Bone Marrow Transplantation advance online publication, 19 May 2014; doi:10.1038/bmt.2014.105.
    Bone marrow transplantation. 05/2014;
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    ABSTRACT: The clinical prognosis of gastrointestinal (GI) graft-versus-host disease (GvHD) is based on either a clinical staging system or histological or endoscopic findings. How these different scores correlate with each other and which have the greater impact on transplantation outcomes is, however, not clear. Clinical, pathological, and endoscopic findings of the upper GI tract on 201 patients who underwent allogeneic hematopoietic stem cell transplantation were reviewed. The association between clinical, histological, and endoscopy grading was assessed by Kendall correlation coefficient. The agreement between grading systems was evaluated by kappa statistics. Factors associated with survival or steroid resistance were analyzed by proportional hazard models. At disease onset, no strong association was found between pathological and clinical grade at disease onset (τ=0.034, P=0.6). In contrast, endoscopy score and clinical grades were strongly associated (τ=0.37, P<0.001). The Kappa concordance coefficient (0.20) between histological and endoscopy scores was poor. However, by multivariate analysis all three scores significantly predicted survival rates Clinical, histological, and endoscopic scores poorly correlated with each other when estimated at the GI-GvHD onset. However, all three severe initial scores independently predict poor outcome. Of interest, clinical and endoscopic scores predict resistance to steroids while pathological does not.
    Transplantation 05/2014; 97(9):965-71. · 3.78 Impact Factor
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    ABSTRACT: Female donors for male recipients worsen the outcome of allogeneic hematopoietic stem-cell transplantation. We wanted to find out whether a male human leukocyte antigen (HLA)-matched unrelated donor (MUD, 8/8, n=2,014) might be an alternative to a female HLA-identical sibling donor (n=2,656) for male patients with acute leukemia. This is a retrospective analysis from the Acute Leukaemia Working Party of the European Group for Blood and Marrow Transplantation. The relative risk (RR) of acute graft-versus-host disease (GVHD) of grades II to IV was increased in the MUD group with acute myeloid leukemia (AML) (RR, 1.47; P<0.001) and acute lymphoblastic leukemia (ALL) (RR, 1.76; P<0.001). There was no difference in incidence of chronic GVHD and nonrelapse mortality between the two groups. Probability of relapse was lower in the MUD group than in the sibling group in patients with ALL (hazards ratio [HR], 0.75; P=0.04) but not in the AML patients (HR, 0.89; P=0.17). Survival was not different between the groups. Leukemia-free survival (LFS) was also similar in the sibling and MUD groups in patients with AML (HR, 1.01; P=0.81) or ALL (HR, 0.93; P=0.45). Factors significantly associated with reduced LFS included active disease, poor cytogenetics, age, year of hematopoietic stem-cell transplantation, reduced-intensity conditioning, and the use of antithymocyte globulin. Male patients who received grafts from male MUDs demonstrated an increased incidence of acute GVHD and LFS same as when using HLA-identical female donors.
    Transplantation 05/2014; · 3.78 Impact Factor
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    ABSTRACT: Posaconazole (PSC) is currently recommended as primary prophylaxis in neutropenic patients with acute myeloid leukemia (AML) and in allogenic hematopoietic stem cell transplantation (AHSCT) recipients with graft-versus-host-disease (GVHD). Studies focusing on breakthrough invasive fungal disease (IFD) upon PSC prophylaxis show disparate results. In order to evaluate IFD incidence in patients on PSC prophylaxis and identify IFD risk factors, we carried out a retrospective study of all consecutive patients on PP from 01/2007 to 12/2010 in our hospital. Breakthrough IFD were identified from the database of the central pharmacy and the French administrative database (PMSI), registering final medical diagnoses of hospitalized patients. Medical data were reviewed to study proven or probable IFD, according to EORTC/MSG definition. PSC plasma concentrations (PPC) were also retrieved. Poisson models were used for statistical analysis. Two hundred and seventy nine patients received PSC prophylaxis for a median duration of 1.4 months (range 0.2-17.9). Proven (n=6) or probable (n=3) IFD were diagnosed in 9 cases (3.2%). IFD incidence rate per 100 person-month was 1.65 (95% CI 0.79-2.97). IFD were candidemia (Candida glabrata n=2), pulmonary invasive aspergillosis (n=3), disseminated fusariosis (n=2) and pulmonary mucormycosis (n=2). Seven deaths were reported, directly related to IFD in 3 patients (33.3%). First dosage of PPC under 0.3μg/ml was the single significant risk factor for IFD (RR 7.77, 95% CI 1.30-46.5, p=0.025). Breakthrough IFD in patients receiving PSC prophylaxis is rare but associated with a poor outcome. Low PSC plasma concentrations are associated with an increased risk of IFDThis article is protected by copyright. All rights reserved.
    Clinical Microbiology and Infection 05/2014; · 4.58 Impact Factor
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    ABSTRACT: VRI are frequent after HSCT and constitute a potential cause of mortality. We analyzed the incidence, risk factors and prognosis of VRI in a cohort of transplanted patients. More frequent viruses were HCoV and HRV followed by flu-like viruses and adenovirus. Risk factors for death were lymphocytopenia and high steroid dosage.
    Biology of blood and marrow transplantation: journal of the American Society for Blood and Marrow Transplantation 04/2014; · 3.15 Impact Factor
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    ABSTRACT: Non-Hodgkin lymphomas (NHL) disproportionately affect older patients who uncommonly receive allogeneic hematopoietic cell transplantation (HCT). We analyzed CIBMTR data on 1248 patients ≥40 years receiving reduced-intensity conditioning (RIC) or non-myeloablative (NMA) HCT for aggressive (n=668) and indolent (n=580) NHL. Aggressive lymphoma was more frequent in the oldest cohort [(age 40-54) 49% vs. (55-64) 57% vs. (≥65) 67% p=0.0008]; fewer patients ≥65 had prior autografting [26% vs. 24% vs. 9%; p=0.002)]. Rates of relapse, acute and chronic GVHD and non-relapse mortality (NRM) at one year were similar [22%, 95% confidence interval (CI) 19-26%; 27%, 95% CI 23-31%; 34%, 95% CI 24-44%]. Progression-free (PFS) and overall (OS) survival at 3 years was slightly lower in older cohorts [OS:54%, 95% CI 50-58%; 40%, 95% CI 36-44%; 39%, 95% CI 28-50%; p<0.0001]. Multivariate analysis revealed no significant effect of age on acute or chronic GVHD or relapse. Age ≥55 years, Karnofsky performance status <80, and HLA-mismatch adversely impacted NRM, PFS, and OS. Disease status at HCT, but not histologic subtype, worsened NRM, relapse, PFS and OS. Even for patients ≥55 years, OS still approached 40% at 3 years suggesting HCT effects long-term remissions and remains underutilized in qualified older patients with NHL.
    Biology of blood and marrow transplantation: journal of the American Society for Blood and Marrow Transplantation 03/2014; · 3.15 Impact Factor
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    ABSTRACT: I.v. BU is frequently used in the conditioning regimen prior to allogeneic hematopoietic SCT (allo-HSCT); however, overall outcomes, incidence of hepatic sinusoidal obstructive syndrome (SOS) and its risk factors are not well known. With this aim, we performed a study on 257 AML adult recipients. Seattle Criteria were used for diagnosis and classification of SOS. The median age was 44 years. Donors were HLA-identical siblings in 60%, HLA-matched unrelated in 29% and HLA mismatched in 11%. Conditioning regimen was myeloablative in 84% (i.v. BU with CY was the most frequently used regimen) and it was reduced intensity in 16% (i.v. BU associated with fludarabine). Acute and chronic GVHD was observed in 28% and 44%, respectively. Two-year incidence of non-relapse mortality was 16±2% and 2-year leukemia-free survival for patients in CR1, CR2 and non remission at HSCT were 55±4%, 58±7%, and 20±5%, respectively. At 6 months, incidence of SOS was 7.8±2%; and it was severe in eight patients (3%). Factors associated with the occurrence of SOS were: HLA-mismatched donor HSCT (P=0.002) and patients transplanted in non-remission (P=0.002). In conclusion, outcomes of HSCT using i.v. BU are encouraging in this setting, SOS incidence is low and it is influenced by the type of donor and disease status at the time of transplant.Bone Marrow Transplantation advance online publication, 17 February 2014; doi:10.1038/bmt.2014.7.
    Bone marrow transplantation 02/2014; · 3.00 Impact Factor
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    ABSTRACT: Paroxysmal nocturnal hemoglobinuria is a rare, acquired disease associated with hemolytic anemia, bone marrow failure, thrombosis, and, frequently, poor quality of life. The International PNH Registry is a worldwide, observational, noninterventional study collecting safety, effectiveness, and quality-of-life data from patients with a confirmed paroxysmal nocturnal hemoglobinuria diagnosis or detectable paroxysmal nocturnal hemoglobinuria clone, irrespective of treatment. In addition to evaluating the long-term safety and effectiveness of eculizumab in a global population, the registry aims to improve diagnosis, optimize patient management and outcomes, and enhance the understanding of the natural history of paroxysmal nocturnal hemoglobinuria. Here we report the characteristics of the first 1610 patients enrolled. Median disease duration was 4.6 years. Median granulocyte paroxysmal nocturnal hemoglobinuria clone size was 68.1% (range, 0.01%-100%). Overall, 16% of patients had a history of thrombotic events and 14% a history of impaired renal function. Therapies included anticoagulation (31%), immunosuppression (19%), and eculizumab (25%). Frequently reported symptoms included fatigue (80%), dyspnea (64%), hemoglobinuria (62%), abdominal pain (44%), and chest pain (33%). Patients suffered from poor quality of life; 23% of patients had been hospitalized due to paroxysmal nocturnal hemoglobinuria-related complications and 17% stated that paroxysmal nocturnal hemoglobinuria was the reason they were not working or were working less. This international registry will provide an ongoing, valuable resource to further the clinical understanding of paroxysmal nocturnal hemoglobinuria. clinicaltrials.gov identifier: NCT01374360.
    Haematologica 01/2014; · 5.94 Impact Factor

Publication Stats

14k Citations
3,355.44 Total Impact Points

Institutions

  • 2008–2014
    • Paris Diderot University
      Lutetia Parisorum, Île-de-France, France
  • 2013
    • University of Minnesota Duluth
      Duluth, Minnesota, United States
    • Indiana Blood and Marrow Transplantation
      Indianapolis, Indiana, United States
    • CHU de Lyon - Institut d'hématologie et d'oncologie pédiatrique
      Lyons, Rhône-Alpes, France
    • University of Pavia
      Ticinum, Lombardy, Italy
    • Pierre and Marie Curie University - Paris 6
      Lutetia Parisorum, Île-de-France, France
    • Vanderbilt University
      • Department of Medicine
      Nashville, MI, United States
  • 2009–2013
    • Mercy Hospital St. Louis
      San Luis, Missouri, United States
    • Centre Hospitalier Universitaire de Nantes
      Naoned, Pays de la Loire, France
    • Hôpital Saint-Louis (Hôpitaux Universitaires Saint-Louis, Laboisière, Fernand-Widal)
      • Service d’Hématologie Greffe de Moelle
      Lutetia Parisorum, Île-de-France, France
    • Trinity College Dublin
      • Department of Haematology
      Dublin, L, Ireland
    • Nottinghamshire Healthcare NHS Trust
      Nottigham, England, United Kingdom
    • Leiden University Medical Centre
      • Department of Hematology
      Leiden, South Holland, Netherlands
  • 2005–2013
    • Universitätsspital Basel
      • Klinik für Infektiologie & Spitalhygiene
      Bâle, Basel-City, Switzerland
    • Centre Hospitalier Universitaire de Grenoble
      Grenoble, Rhône-Alpes, France
    • Azienda Ospedaliera Universitaria San Martino di Genova
      Genova, Liguria, Italy
    • National Institutes of Health
      • Division of Cancer Epidemiology and Genetics
      Bethesda, MD, United States
  • 2004–2013
    • French Institute of Health and Medical Research
      Lutetia Parisorum, Île-de-France, France
  • 2012
    • University of Patras
      Rhion, West Greece, Greece
    • Sheba Medical Center
      Gan, Tel Aviv, Israel
    • Université Paris-Sorbonne - Paris IV
      Lutetia Parisorum, Île-de-France, France
    • University of Michigan
      • Comprehensive Cancer Center
      Ann Arbor, MI, United States
    • Hospital de la Santa Creu i Sant Pau
      Barcino, Catalonia, Spain
    • University Hospital Estaing of Clermont-Ferrand
      Clermont, Auvergne, France
    • Karolinska University Hospital
      Tukholma, Stockholm, Sweden
    • University of Liège
      Luik, Walloon Region, Belgium
    • King's College London
      Londinium, England, United Kingdom
  • 2011–2012
    • National Marrow Donor Program
      Minneapolis, Minnesota, United States
    • Klinikum Augsburg
      Augsberg, Bavaria, Germany
    • University of Florida
      • College of Medicine
      Gainesville, FL, United States
  • 2010–2012
    • University of Minnesota Twin Cities
      • • Division of Hematology, Oncology and Transplantation
      • • Department of Pediatrics
      Minneapolis, MN, United States
    • University of Nantes
      Naoned, Pays de la Loire, France
    • Hôpital Européen Georges-Pompidou (Hôpitaux Universitaires Paris-Ouest)
      Lutetia Parisorum, Île-de-France, France
    • Azienda Ospedaliera San Camillo Forlanini
      Roma, Latium, Italy
    • Asklepios Klinik St. Georg
      Hamburg, Hamburg, Germany
  • 2009–2012
    • Universitätsklinikum Freiburg
      Freiburg an der Elbe, Lower Saxony, Germany
  • 2004–2011
    • Assistance Publique – Hôpitaux de Paris
      Lutetia Parisorum, Île-de-France, France
  • 2004–2010
    • Unité Inserm U1077
      Caen, Lower Normandy, France
  • 2007–2009
    • Hôpital Henri Mondor (Hôpitaux Universitaires Henri Mondor)
      • Service d’Hématologie Clinique
      Créteil, Ile-de-France, France
    • Centre Hospitalier Intercommunal Creteil
      Créteil, Île-de-France, France
    • Azienda Ospedaliera San Paolo - Polo Universitario
      Milano, Lombardy, Italy
    • University of the West of England, Bristol
      • Department of Applied Sciences
      Bristol, ENG, United Kingdom
    • Università degli Studi di Milano-Bicocca
      Milano, Lombardy, Italy
  • 1998–2008
    • Fred Hutchinson Cancer Research Center
      • Division of Clinical Research
      Seattle, Washington, United States
  • 2006
    • University of Utah
      • Division of Rheumatology
      Salt Lake City, UT, United States
    • Showa University
      • Department of Hematology
      Shinagawa, Tōkyō, Japan
  • 1999–2006
    • Medical College of Wisconsin
      • Center for International Blood & Marrow Transplant Research
      Milwaukee, WI, United States
    • IRCCS Istituto G. Gaslini
      Genova, Liguria, Italy
    • St. George's School
      • Division of Haematology
      Middletown, Rhode Island, United States
  • 2002
    • Hospital Clínic de Barcelona
      Barcino, Catalonia, Spain
    • Institute Mutualiste Montsouris
      Lutetia Parisorum, Île-de-France, France
  • 2001–2002
    • Institut Universitaire de France
      Lutetia Parisorum, Île-de-France, France
  • 1988–1997
    • Institut de Cancérologie Gustave Roussy
      • Department of Radiotherapy
      Île-de-France, France
  • 1995
    • Institut Curie
      • Service de Radiothérapie
      Lutetia Parisorum, Île-de-France, France
  • 1992
    • University of Texas MD Anderson Cancer Center
      • Department of Biomathematics
      Houston, TX, United States