Defibrotide for prophylaxis of hepatic veno-occlusive disease in paediatric haemopoietic stem-cell transplantation: an open-label, phase 3, randomised controlled trial

Corbacioglu, Selim; Cesaro, Simone; Faraci, Maura; Valteau‐Couanet, Dominique; Gruhn, Bernd; Rovelli, Attilio; Boelens, Jaap Jan; Hewitt, Annette; Schrum, Johanna; Schulz, Ansgar; Müller, Ingo; Stein, Jerry; Wynn, Robert F.; Greil, Johann; Sykora, Karl Walter; Matthes‐Martin, Susanne; Führer, Monika; O’Meara, Anne; Toporski, Jacek; Sedláček, Petr; Schlegel, Paul G.; Ehlert, Karoline; Fasth, Anders; Winiarski, Jacek; Arvidson, Johan; Mauz‐Körholz, Christine; Özşahin, Hülya; Schrauder, André; Bader, Peter; Massaro, Joseph M.; D’Agostino, Ralph B.; Hoyle, Margaret; Iacobelli, Massimo; Debatin, Klaus‐Michael; Peters, Christina; Dini, Giorgio

doi:10.1016/s0140-6736(11)61938-7

Cited by 328 publications

(382 citation statements)

References 34 publications

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“…22 Since 2005, defibrotide has been administered (25 mg/kg/day IV in four doses) as prophylaxis against sinusoidal occlusion syndrome (SOS). 23 Since 1987, patients are isolated in single laminar airflow rooms until resolution of complications. All patients received trimethoprim-sulfamethoxazole as Pneumocystis Jiroveci prophylaxis.…”

Section: Hd Chemotherapymentioning

confidence: 99%

Busulfan–melphalan in high-risk neuroblastoma: the 30-year experience of a single institution

Pasqualini

Blanchard

et al. 2016

Bone Marrow Transplant

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High-dose chemotherapy (HDC) was investigated in high-risk neuroblastoma (HR-NBL) to reduce the risk of relapse. We report the results of the 30-year experience of a cohort of patients with HR-NBL treated with high-dose (HD) busulfan (Bu)-containing regimens. From 1980From to 2009 patients aged 41 year with stage 4 NBL were treated with HD Bu-containing regimens at Gustave Roussy. These data were prospectively recorded in the Pediatric Transplantation Database. The median age at diagnosis was 40 months (12-218 months). All patients had a stage 4 neuroblastoma. NMYC amplification was displayed in 24% of the tumors. The hematopoietic support consisted of bone marrow or PBSCs in 46% and 49% of patients, respectively. The 5-year eventfree survival and overall survival rates of the whole cohort were 35.1% and 40%, respectively. Age at diagnosis, bone marrow involvement and tumor response after induction chemotherapy were significant prognostic factors. Toxicity was manageable and decreased over time, owing to both PBSC administration and better supportive care. Based on this experience, HD Bu-melphalan (Mel) has been implemented in Europe and compared with Carboplatin-Etoposide-Mel in the European SIOP Neuroblastoma (SIOPEN)/HR-NBL randomized protocol. It has now become the standard HDC in the SIOPEN HR strategy. INTRODUCTIONThe improved survival rates of patients with high-risk (HR) neuroblastoma (NBL) observed during the past decades are related to the intensification of induction therapy, high-dose chemotherapy (HDC) and better supportive care. 1 In the HR-NBL population, a correlation between survival and dose intensity has been reported, and three randomized studies showed that HDC followed by autologous stem cell transplantation (ASCT) or autologous bone marrow transplantation (ABMT) improved survival rates. [2][3][4] As NBL is notoriously radiosensitive, hyperfractionated TBI was incorporated into the conditioning regimen and has yielded encouraging results. 5 Alkylating agents were demonstrated to produce a log-linear dose response against tumor cells in vitro. 6 Since the mid-1980s, HDC with alkylating agents and ASCT has been the treatment strategy used at Gustave Roussy. HD busulfan (Bu) was selected because of its specific cytotoxicity against quiescent cells, 7 as an alternative to TBI in HR-NBL patients to avoid its side-effects, 8 especially in this very young population, and to achieve better survival rates.We report here the results of our 30-year experience on patients with HR-NBL treated with HD Bu-containing regimens. Bu was combined with melphalan (Mel) or Mel plus cyclophosphamide, and HDC was followed by stem cell (SC) or bone marrow (BM) transplantation. These results provided the rationale to widely implement the use of HD Bu-Mel, which was then compared with Carboplatin-Etoposide-Mel (CEM) in the European SIOP Neuroblastoma (SIOPEN)/HR-NBL randomized trial. HD Bu-Mel has now become the standard HD regimen in the SIOPEN HR strategy.

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Section: Hd Chemotherapymentioning

confidence: 99%

Busulfan–melphalan in high-risk neuroblastoma: the 30-year experience of a single institution

Pasqualini

Blanchard

et al. 2016

Bone Marrow Transplant

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“…Defibrotide was administered (25 mg/kg per day IV in four doses) as prophylaxis against sinusoidal obstruction syndrome (SOS) since 2006. 17 Acyclovir prophylaxis was not administered (Guerrini-Rousseau L, SIOP meeting 2014; P-0158). All patients received trimethoprim-sulfamethoxazole as prophylaxis against Pneumocystis jiroveci.…”

Section: Supportive Carementioning

confidence: 99%

“…CR and PR were obtained in 3/26 and 23/26 patients, respectively. The mIBG scan was positive in 18/26 children, with a median SIOPEN score of 2 (range [1][2][3][4][5][6][7][8][9][10][11][12][13][14][15][16][17][18][19][20]. Three patients still exhibited hepatic metastasis, and seven patients had a positive bone marrow biopsy.…”

Section: Patient Characteristicsmentioning

confidence: 99%

Tandem high-dose chemotherapy with thiotepa and busulfan–melphalan and autologous stem cell transplantation in very high-risk neuroblastoma patients

Pasqualini

Dufour

Goma

et al. 2015

Bone Marrow Transplant

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“…Interpretation: Defibrotide prophylaxis seems to reduce incidence of veno-occlusive disease and is well tolerated. Thus, such prophylaxis could present a useful clinical option for this serious complication of HSCT [21] .…”

Section: Introductionmentioning

confidence: 99%

Putative Prophylaxes Updated of Placenta Extract and Aloe vera as Biogenic Stimulants

Yagi

Ataka

2014

Journal of Gastroenterology and Hepatology Research

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Gastroenterology and Hepatology Researchdation, and the possible putative prophylaxes to optimize cognitive decline in older adults with pre-diabetes symptom. Because of the diversities of placental extract and Aloe vera as biogenic stimulants, present review introduced the briefly selected authors' intentions in each topical subject. Specially, the influence of gut microbiota to host is astonishing and highlighting that the consequence of our behaviours affect not only the environment without, but also that within us. Based on geriatric and neurological viewpoint, present review highlights current therapeutic strategies of placenta extract and Aloe vera as the so-called "biogenic stimulants", aimed at producing a biological rejuvenator in placental extract and improving cognitive function via modifying the brain's insulin sensitivity in Aloe vera.

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Defibrotide for prophylaxis of hepatic veno-occlusive disease in paediatric haemopoietic stem-cell transplantation: an open-label, phase 3, randomised controlled trial

Cited by 328 publications

References 34 publications

Busulfan–melphalan in high-risk neuroblastoma: the 30-year experience of a single institution

Busulfan–melphalan in high-risk neuroblastoma: the 30-year experience of a single institution

Tandem high-dose chemotherapy with thiotepa and busulfan–melphalan and autologous stem cell transplantation in very high-risk neuroblastoma patients

Putative Prophylaxes Updated of Placenta Extract and Aloe vera as Biogenic Stimulants

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