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Acute GvHD Incidence and Outcome: Single Center Experience
Abstracts / Biol Blood Marrow Transplant 23 (2017) S18–S391
range- suggesting that BMT course may have a greater affect on the need for IVIG than conditioning regimen, diagnosis, or donor source. One third of the patients did not receive any IVIG, and the at group was heterogeneous for conditioning regimen, diagnosis, and donor source. Most allogeneic recipients had a normal B-Cell count at the first time point tested suggesting that our standard time points may not capture data on early B-Cell recovery.
307 Acute GvHD Incidence and Outcome: Single Center Experience Eman M. Elsabbagh 1,2, Stacey Shubert 1, Kathryn Leung 1, Swati Naik 1, Stephen Gottschalk 1, Carl Allen 1, Khaled Yassine 1, Bilal Omer 1, Nabil Ahmed 1, Ghadir Sasa 1, Meena Hegde 1, Malcolm K. Brenner 1, Helen E. Heslop 1, Ann M. Leen 3, Robert A. Krance 1, Caridad Martinez 1. 1 Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children’s Hospital, Houston, TX; 2 Department of Pediatrics, Division of Hematology & Oncology, Mansoura University Children’s Hospital, Mansoura Medical School, Mansoura University, Mansoura, Egypt; 3 Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children’s Hospital, Houston Methodist Hospital, Houston, TX Acute graft vs host disease (aGvHD) remains a major cause of post-transplantation treatment-related mortality (TRM). aGvHD affects up to 50% of SCT recipients even with the administration of standard prophylactic immunosuppressive regimens; thus it remains an important posttransplantation complication, leading to high morbidity and mortality in allogeneic SCT recipients. Here we report our single center incidence of aGvHD from 2011-2013 in 148 patients with a median age of 8 years (range, .5-23 years) with: hematological disease (n = 28), acute leukemia (n = 58), MDS (n = 9), lymphoma (n = 15), HLH (n = 14), immunodeficiency (n = 22), EBV related lymphoproliferative disease (n = 1) and metabolic disorder (n = 1). Donors included 69 MUD/MMUD, 48 MRD/MMRD, 13 UCB, and 18 haplo. Campath was administered in most conditioning regimen (123/148). Sixty-one patients had aGvHD (41%) at a median time of 67 days (range, .5-18 m) after transplant with only 14% being severe aGVHD (21/148). Among the patients with aGvHD, twenty-one received a related donor matched or mismatched, MUD/MMUD (29), haplo (4), and UCB (7). aGvHD involved skin only for 38 pts, gut only for 3 pts, skin and liver for 9 pts, skin and gut 7 pts; skin, gut, and liver for 4 pts. Thirty-six patients developed stage II-IV aGvHD with 21 being stage III-IV aGVHD. 62% (13/21) of severe aGvHD was noted on the MUD/MMUD recipients. Overall survival of the whole cohort was 77% (115/148) with 78% (48/61) survival among the patients with aGvHD. The causes of death among the aGVHD patients were: GvHD (n = 3), pulmonary hemorrhage (n = 2), infection (n = 2), disease relapse (n = 3), and multi-organ failure (n = 3). In conclusion aGvHD is a common occurrence after pediatric SCT but only 13 patients developed severe grade III-IV which is possibly secondary to younger age and use of campath. Interestingly patients with aGvHD had similar overall survival when compared to patients without GvHD.
S231
308 Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease in a Single Institution in Brazil. Reproducing Good Results with a Reduced Toxicity Regimen Juliana Folloni Fernandes 1, Luiz Fernando Alves Lima Mantovani 1, Angela Mandelli Venancio 1, Mayra Dorna 1, Antonio Carlos Pastorino 1, Dewton Vasconcelos 2, Antonio Condino Neto 3, Ana Carla Augusto Moura 4, Maria Dulce Collassanti 1, Maria Aparecida Zanichelli 1, Magda Carneiro-Sampaio 1, Vanderson G. Rocha 2, Vicente Odone Filho 1. 1 ITACI—Instituto da Criança, Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo, São Paulo, Brazil; 2 Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo, São Paulo, Brazil; 3 Universidade de São Paulo, São Paulo, Brazil; 4 Instituto de Medicina Integral Professor Fernando Figueira, Recife, Brazil Hematopoietic stem cell transplantation (HSCT) is a curative alternative for patients with Chronic Granulomatous Disease (CGD), a primary immunodeficiency (PID) characterized by an inherited defect of the neutrophil function which can lead to life threatening infections and granulomas. Historically, HSCT with conventional myeloablative conditioning regimens has been associated with high toxicity for patients with CGD. More recently, International Working Groups on PID have published an experience with reduced toxicity regimen showing important improvements in transplant outcomes for these patients. We describe here the experience of our center with a reduced toxicity regimen for patients with CGD. Methods: We conducted a retrospective analysis of nine patients who underwent allogeneic HSCT for Chronic Granulomatous Disease at the Children’s Institute of the University of São Paulo, using a reduced toxicity regimen. Results: From 2010 to May 2016, nine patients with CGD were submitted to HSCT in our center. Risk features pre-HSCT included: pulmonary aspergillosis, pulmonary tuberculosis, staphylococcal abscess, disseminated BCGosis, most having multiple severe infections and granulomas. Three of the patients had active infection at the moment of transplant. Median age at transplant was 7 years (range 1-14). Donors were: siblings (n = 3) or matched unrelated donors (MUD) (n = 6), considering HLA A, B, C and DRB1 (high resolution). Graft source was bone marrow for all 9 patients. All patients received the same preparative regimen consisting of: fludarabine 180 mg/m2, intravenous bussulfan (AUC range 45-65 mg/l*h) and either antithymocyte globulin (ATG) 7,5 mg/Kg (n = 7) or alemtuzumab 0,6 mg/Kg (n = 2). Graft versus host disease (GVHD) prophylaxis consisted of cyclosporine A and mycophenolate. Post-transplantation complications included: mild mucositis (n = 6), acute GVHD grade II-III (n = 5; 4 = only skin and 1 = skin and gut), and infections (CMV, n = 3; BKvirus, n = 1; sepsis, n = 1). One patient developed limited chronic GVHD of skin and mouth. All patients engrafted, with predominantly donor chimerism. One patient had loss of chimerism 8 months after HSCT and was successfully submitted to a second transplant with the same sibling donor. One patient died 50 days after transplant of sepsis. Eight patients are alive and well, at a median follow-up of 19 (range 4-57) months post-transplantation. Conclusion: Allogeneic HSCT with reduced toxicity regimen is a good alternative in the treatment of patients with CGD. These good results may expand the indications of transplant in this disease, including patients with less severe complications pre HSCT.
range- suggesting that BMT course may have a greater affect on the need for IVIG than conditioning regimen, diagnosis, or donor source. One third of the patients did not receive any IVIG, and the at group was heterogeneous for conditioning regimen, diagnosis, and donor source. Most allogeneic recipients had a normal B-Cell count at the first time point tested suggesting that our standard time points may not capture data on early B-Cell recovery.
307 Acute GvHD Incidence and Outcome: Single Center Experience Eman M. Elsabbagh 1,2, Stacey Shubert 1, Kathryn Leung 1, Swati Naik 1, Stephen Gottschalk 1, Carl Allen 1, Khaled Yassine 1, Bilal Omer 1, Nabil Ahmed 1, Ghadir Sasa 1, Meena Hegde 1, Malcolm K. Brenner 1, Helen E. Heslop 1, Ann M. Leen 3, Robert A. Krance 1, Caridad Martinez 1. 1 Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children’s Hospital, Houston, TX; 2 Department of Pediatrics, Division of Hematology & Oncology, Mansoura University Children’s Hospital, Mansoura Medical School, Mansoura University, Mansoura, Egypt; 3 Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children’s Hospital, Houston Methodist Hospital, Houston, TX Acute graft vs host disease (aGvHD) remains a major cause of post-transplantation treatment-related mortality (TRM). aGvHD affects up to 50% of SCT recipients even with the administration of standard prophylactic immunosuppressive regimens; thus it remains an important posttransplantation complication, leading to high morbidity and mortality in allogeneic SCT recipients. Here we report our single center incidence of aGvHD from 2011-2013 in 148 patients with a median age of 8 years (range, .5-23 years) with: hematological disease (n = 28), acute leukemia (n = 58), MDS (n = 9), lymphoma (n = 15), HLH (n = 14), immunodeficiency (n = 22), EBV related lymphoproliferative disease (n = 1) and metabolic disorder (n = 1). Donors included 69 MUD/MMUD, 48 MRD/MMRD, 13 UCB, and 18 haplo. Campath was administered in most conditioning regimen (123/148). Sixty-one patients had aGvHD (41%) at a median time of 67 days (range, .5-18 m) after transplant with only 14% being severe aGVHD (21/148). Among the patients with aGvHD, twenty-one received a related donor matched or mismatched, MUD/MMUD (29), haplo (4), and UCB (7). aGvHD involved skin only for 38 pts, gut only for 3 pts, skin and liver for 9 pts, skin and gut 7 pts; skin, gut, and liver for 4 pts. Thirty-six patients developed stage II-IV aGvHD with 21 being stage III-IV aGVHD. 62% (13/21) of severe aGvHD was noted on the MUD/MMUD recipients. Overall survival of the whole cohort was 77% (115/148) with 78% (48/61) survival among the patients with aGvHD. The causes of death among the aGVHD patients were: GvHD (n = 3), pulmonary hemorrhage (n = 2), infection (n = 2), disease relapse (n = 3), and multi-organ failure (n = 3). In conclusion aGvHD is a common occurrence after pediatric SCT but only 13 patients developed severe grade III-IV which is possibly secondary to younger age and use of campath. Interestingly patients with aGvHD had similar overall survival when compared to patients without GvHD.
S231
308 Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease in a Single Institution in Brazil. Reproducing Good Results with a Reduced Toxicity Regimen Juliana Folloni Fernandes 1, Luiz Fernando Alves Lima Mantovani 1, Angela Mandelli Venancio 1, Mayra Dorna 1, Antonio Carlos Pastorino 1, Dewton Vasconcelos 2, Antonio Condino Neto 3, Ana Carla Augusto Moura 4, Maria Dulce Collassanti 1, Maria Aparecida Zanichelli 1, Magda Carneiro-Sampaio 1, Vanderson G. Rocha 2, Vicente Odone Filho 1. 1 ITACI—Instituto da Criança, Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo, São Paulo, Brazil; 2 Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo, São Paulo, Brazil; 3 Universidade de São Paulo, São Paulo, Brazil; 4 Instituto de Medicina Integral Professor Fernando Figueira, Recife, Brazil Hematopoietic stem cell transplantation (HSCT) is a curative alternative for patients with Chronic Granulomatous Disease (CGD), a primary immunodeficiency (PID) characterized by an inherited defect of the neutrophil function which can lead to life threatening infections and granulomas. Historically, HSCT with conventional myeloablative conditioning regimens has been associated with high toxicity for patients with CGD. More recently, International Working Groups on PID have published an experience with reduced toxicity regimen showing important improvements in transplant outcomes for these patients. We describe here the experience of our center with a reduced toxicity regimen for patients with CGD. Methods: We conducted a retrospective analysis of nine patients who underwent allogeneic HSCT for Chronic Granulomatous Disease at the Children’s Institute of the University of São Paulo, using a reduced toxicity regimen. Results: From 2010 to May 2016, nine patients with CGD were submitted to HSCT in our center. Risk features pre-HSCT included: pulmonary aspergillosis, pulmonary tuberculosis, staphylococcal abscess, disseminated BCGosis, most having multiple severe infections and granulomas. Three of the patients had active infection at the moment of transplant. Median age at transplant was 7 years (range 1-14). Donors were: siblings (n = 3) or matched unrelated donors (MUD) (n = 6), considering HLA A, B, C and DRB1 (high resolution). Graft source was bone marrow for all 9 patients. All patients received the same preparative regimen consisting of: fludarabine 180 mg/m2, intravenous bussulfan (AUC range 45-65 mg/l*h) and either antithymocyte globulin (ATG) 7,5 mg/Kg (n = 7) or alemtuzumab 0,6 mg/Kg (n = 2). Graft versus host disease (GVHD) prophylaxis consisted of cyclosporine A and mycophenolate. Post-transplantation complications included: mild mucositis (n = 6), acute GVHD grade II-III (n = 5; 4 = only skin and 1 = skin and gut), and infections (CMV, n = 3; BKvirus, n = 1; sepsis, n = 1). One patient developed limited chronic GVHD of skin and mouth. All patients engrafted, with predominantly donor chimerism. One patient had loss of chimerism 8 months after HSCT and was successfully submitted to a second transplant with the same sibling donor. One patient died 50 days after transplant of sepsis. Eight patients are alive and well, at a median follow-up of 19 (range 4-57) months post-transplantation. Conclusion: Allogeneic HSCT with reduced toxicity regimen is a good alternative in the treatment of patients with CGD. These good results may expand the indications of transplant in this disease, including patients with less severe complications pre HSCT.