Universidade Federal de São Paulo, Inst Oncol Pediat, GRAACC, São Paulo, BrazilUniversidade Federal de São Paulo, Inst Oncol Pediat, GRAACC, São Paulo, BrazilWeb of Scienc

Barros, Daniele Port

Caran, E. M.

Félix, Olga Margareth Wanderley de Oliveira

Ginani, Valéria Cortez

Gouveia, Roseane Vasconcelos

Ibanez, A.

Lederman, H. M.

Lee, Maria Lúcia de Martino

Marconcini, J.

Petrilli, A. A.

Seber, Adriana

Seixas, M. T.

Simoes, P. C.

Villela, N.

Zecchin, Victor Gottardello

|Macedo, Carla Renata Pacheco Donato

Biology of Blood and Marrow Transplantation

Seber, Adriana [UNIFESP]

Ginani, Valéria Cortez [UNIFESP]

Gouveia, Roseane Vasconcelos [UNIFESP]

Zecchin, Victor Gottardello [UNIFESP]

Barros, Daniele Port [UNIFESP]

Ibanez, A. [UNIFESP]

Marconcini, J. [UNIFESP]

Villela, N. [UNIFESP]

Félix, Olga Margareth Wanderley de Oliveira [UNIFESP]

Simoes, P. C. [UNIFESP]

Lee, Maria Lúcia de Martino [UNIFESP]

Lederman, H. M. [UNIFESP]

Caran, E. M. [UNIFESP]

|Macedo, Carla Renata Pacheco Donato [UNIFESP]

Petrilli, A. A. [UNIFESP]

Repositório Institucional UNIFESP

S264 Poster Session Isignificant morbidity and mortality. Factors likely include pathogen-esis of the disease as well as prolonged prior exposure to cytotoxicchemotherapy and immune suppression. Clinical approaches to im-prove outcomes should optimize disease control while minimizingtoxicity before and after transplant.302IMPROVED SURVIVAL FOLLOWING HLA-MATCHED RELATED MARROWTRANSPLANTATION IN PEDIATRIC PATIENTS WITH SEVERE APLASTICANEMIA: (A 39-YEAR RETROSPECTIVE ANALYSIS)Burroughs, L.1,2, Woolfrey, A.1,2, Storer, B.1,3, Deeg, H.J.1,2,Flowers, M.1,2, Martin, P.1,2, Carpenter, P.1,2, Doney, K.1,2,Appelbaum, F.1,2, Sanders, J.1,2, Storb, R.1,2 1Fred Hutchinson CancerResearch Center; 2University ofWashington School of Medicine; 3Univer-sity of Washington School of Public HealthAllogeneic marrow transplantation offers curative therapy forpediatric patients with severe aplastic anemia. Over the past 4 de-cades, significant improvements in the long term outcome of pa-tients treated with allogeneic marrow grafts have resulted fromprogress in the prevention and treatment of graft rejection andgraft-versus-host disease (GVHD). Here we report the outcomeof 149 pediatric patients, ages 2-19 years who received HLA-matched related marrow grafts for treatment of severe aplastic ane-mia between 1971 and 2009. Patients were divided into 3 groups,reflecting changes in conditioning and GVHD prophylaxis regi-mens that occurred over time. Group 1 consisted of 100 patientswho were conditioned with Cyclophosphamide (CY; 200 mg/kg)followed by ‘‘long’’ (102 days) Methotrexate (MTX) for GVHDprevention. Group 2 consisted of 19 patients who received CY fol-lowed by ‘‘short’’ (days 1, 3, 6, and 11) MTX and cyclosporine(CSP; through day 180). Group 3 consisted of 30 patients whowere conditioned with CY and horse antithymocyte globulin(ATG) followed by MTX and CSP for GVHD prevention. Therisk of mortality was significantly different between the 3 groups(p\0.0001).With a median follow up of 25.2 (range, 0.3-37) years,the 5-year survival estimates were 66% for group 1, 95% for group2, and 100% for group 3. There was a suggestion that the risk of re-jection was different between the 3 groups (p5 0.06) and the 3-yearestimates of graft rejection were 22%, 32%, and 12%, respectively.The estimated probabilities of grades III-IV acute GVHD were15% for group 1, 0% for group 2, and 3% for group 3. The2-year estimates of chronic GVHD were 21%, 21%, and 7%,respectively. In summary, advances in conditioning and GVHDprophylaxis regimens as well as supportive care during the past 39years have led to improved outcomes for pediatric patients withsevere aplastic anemia. These results strongly support the use ofallogeneic marrow transplantation for newly diagnosed pediatricpatients with severe aplastic anemia who have an HLA-matchedrelated donor.303HIGH DOSE ORAL BUSULFAN AND INTRAVENOUS MELPHALAN ASCONDITIONING THERAPY FOR AUTOLOGOUS HEMATOPOIETIC STEMCELL TRANSPLANT (HSCT) FOR THE TREATMENT OF PEDIATRIC SOLIDTUMORSSeber, A.1, Ginani, V.C.1, Gouveia, R.V.1, Zecchin, V.G.1, Barros, D.P.1,Ibanez, A.1, Marconcini, J.1, Villela, N.1, Felix, O.M.W.O.1,Simoes, P.C.1, Seixas, M.T.2, Lee, M.L.M.1, Lederman, H.M.1,Caran, E.M.1, Macedo, C.R.1, Petrilli, A.A.1 1 Instituto de Oncologia+ TBI Focal radiation n (%)n (%) SMN in field n (% with focal)Cases with SMN (n56) 5 (83%) 5 (83%)2/5 (40%)Controls with SMN (n54) 3 (75%) 2 (50%)1/2 (50%)Pediatrica - GRAACC - Unifesp, S~ao Paulo, SP, Brazil; 2UniversidadeFederal de S~ao Paulo, S~ao Paulo, SP, BrazilDisseminated or relapsed pediatric solid tumors usually have dis-mal prognosis and HSCT has been used as a treatment option forchildren with chemo-sensitive tumors. With modern intensive pro-tocols, patients usually have been treated with many different drugsand may have some degree of renal or cardiac compromise. One in-teresting alternative would be to use two alkylating agents, busulfanand melphalan: these drugs are not used in pediatric chemotherapyprotocols, their most important dose-limiting toxicity is marrowsuppression, and they are not toxic to the heart or kidneys. Our ob-jective is to describe the institutional experience with autologousHSCTwith busulfan-melphalan for the treatment of relapsed or dis-seminated pediatric solid tumors.Results: Nineteen patients with a median age of 11 years (2-33), 10male, were transplanted, 12/19 with measurable disease. Diagnoseswere alveolar sarcoma (1), desmoplastic small round cell tumor(DSRCT, 1), Wilms tumor (1), primitive neuroectodermal tumor(PNET, 2), synovial sarcoma (2), Ewing sarcoma (6, all but 1 withadvanced disease), and stage-4 neuroblastoma (6, only 1 in 1st remis-sion). All patients had normal marrow aspirate and biopsy prior totransplant, including immunocytochemistry. Conditioning therapywas busulfan 1 mg/kg/dose q 6 hours PO for 4 days on D-5 to D-2 and melphalan 140 mg/m2 IV on D-1. Local irradiation, whenplanned, was postponed for the post transplant period. Three re-ceived bone marrow grafts and 16 peripheral blood stem cells withtarget cell dose of 5x106 CD34 cells/kg. All patients engraftedpromptly and none of them had severe toxicities. One had late sinu-soidal obstruction syndrome and one Ewing sarcoma patient hadGuillain Barre Syndrome at the time of post HSCT irradiation tohis leg. One patient with PNET, the patient with DSRCT, Wilmstumor and 3/6 with Ewing sarcoma died of their primary disease af-ter a median of 320 days post HSCT. The patient with alveolar sar-coma, both with synovial sarcoma, 3 with Ewing sarcoma and allneuroblastoma patients remain in remission and well after a medianof 10.5 months post transplant (2-119). In conclusion, busulfan-mel-phalan was very well tolerated and 13/19 patients with advanced tu-mors remain well and in remission. Longer follow up and a largernumber of patients are needed to define the – promising – role ofthis preparative regime for high dose chemotherapy and HSCT inthe treatment of relapsed or disseminated solid tumors.304INCIDENCE OF SECOND MALIGNANCIES (SMN) IN PEDIATRIC HEMATO-POIETIC STEM CELL TRANSPLANT (HSCT) RECIPIENTS: A REPORT COM-PARING THOSE WITH AND WITHOUT EXOSTOSESDanner-Koptik, K.1, Kletzel, M.1,2, Dilley, K.J.1,2 1Children’s MemorialHospital, Chicago, IL; 2Northwestern University, Chicago, ILAmong 650 patients transplanted in our institution between 1992and 2008, 19 (3%) are known to have developed a SMNpost-HSCT.We have studied HSCT survivors who developed exostoses. Weidentified 27 patients with clinical and/or radiologic exostoses trans-planted between 3/1992 and 12/2003. One patient with exostosis hadco-existent malignancies and was treated elsewhere so was excludedfrom all analyses due to incomplete data and the possibility of a ge-netic cancer susceptibility. This group was compared to controls thatwere matched for gender, age within 3 years and on same side of pu-berty, malignancy/not and HSCT type. Our aim is to describewhether the exostosis patients have a higher risk for developingSMNs post-HSCT. There were 6/26 (23%) with SMNs in the exos-tosis group, 4/26 (15%) in the control group (McNemar p-valueAge at first HSCT Time HSCT to SMN A/C GvHDMean (range) Mean (range) %5.5y (0.5-9.1) 11.3y (6.6-16.1) AGvHD 17%CGvHD 50%4.9y (1.4-9.1) 9.0y (4.3-13.3) AGvHD 75%CGvHD 25%

HIGH DOSE ORAL BUSULFAN and INTRAVENOUS MELPHALAN AS CONDITIONING THERAPY for AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANT (HSCT) for the TREATMENT of PEDIATRIC SOLID TUMORS

HIGH DOSE ORAL BUSULFAN and INTRAVENOUS MELPHALAN AS CONDITIONING THERAPY for AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANT (HSCT) for the TREATMENT of PEDIATRIC SOLID TUMORS

Abstract

Similar works

Full text

Available Versions

Repositório Institucional UNIFESP