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A Prospective Phase III Trial for Comparing Cyclophosphamide and Fludarabine Conditioning Regimen in Severe Idiopathic Aplastic Anemia
Abstracts / Biol Blood Marrow Transplant 26 (2020) S96 S255
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371 Long Term Outcomes of Patients with Aggressive T-Cell Non-Hodgkin Lymphoma Undergoing Allogeneic Stem Cell Transplantation: Retrospective Results from Single Center. Amandeep Salhotra MD1, Liana Nikolaenko MD2, Lu Chen PhD3, Ni-Chun Tsai MS, MPH4, D. Lynne Smith PhD2, Auayporn Nademanee MD5, Leslie Popplewell MD, FACP5, Alex F. Herrera MD2, Matthew Mei MD5, Stephen J. Forman MD5, Jasmine Zain MD5. 1 Hematology/Hematopoietic Cell Transplantation, City of Hope National Medical Center, Duarte, CA; 2 Hematology and Hematopoietic Cell Transplantation, City of Hope National Medical Center, Duarte, CA; 3 Computational and Quantitative Medicine, City of Hope National Medical Center, Duarte, CA; 4 Computational and Quantitative Medicine-BRI, City of Hope National Medical Center, Duarte, CA; 5 Hematology/ Hematopoietic Cell Transplant, City of Hope National Medical Center, Duarte, CA Introduction: Peripheral T-cell lymphomas (PTCL) comprise 1520% of adult non-Hodgkin lymphoma and have a poor prognosis; 5-year survival is less than 30% for the most aggressive subtypes. Allogeneic HCT (allo-HCT) is offered to eligible patients as a potentially curative modality in the salvage setting or in high risk patients to consolidate an initial response to frontline therapy. Objective: To report clinical outcomes derived from large sample size and long-term follow up data. Methods: We retrospectively reviewed medical records of 87 consecutive patients with PTCL who underwent allo-HCT at City of Hope from January 2000 to June 2018. Baseline patient demographic, treatment, and disease characteristics were summarized by descriptive statistics. Overall survival (OS) and progressionfree survival (PFS) were evaluated by Kaplan-Meier curves and the log-rank test. Cumulative incidences of time to relapse and time to non-relapse mortality (NRM) were calculated with relapse and NRM as competing risks. Cumulative incidences of acute and chronic GVHD were calculated as time to onset of GVHD with relapse and death as competing events for GVHD. Results: Median age at allo-HCT was 49 (range 2-70) years. Histologies were PTCL-NOS (n=21); transformed CTCL (n=19); NK TCL (n=17); AITL (n=15), ALCL (n=7); gdTCL (n=6) and other rare subtypes (n=2). No patients had a prior auto transplant. 42 patients (48%) had myeloablative conditioning; FTBI-based (n=39), or BEAM regimen (n=3). 45 patients (52%) had reduced intensity conditioning with a fludarabine/melphalan based regimen in 39. Sibling HCT was done in 47 (54%) patients. MUD HCT was done in 36 (41%); donors were fully HLA matched for 15 (17%) patients and mismatched in 21 (24%); 4 (5%) got haploidentical HCT. The most common GVHD prophylaxis was tacrolimus/sirolimus (n=54). Stem cell source was PBSC in 77 (88%), bone marrow in 5 (6%), and cord blood in 5 (6%) patients. At allo-HCT 25 (29%) patients were in complete remission, 25 (29%) in partial remission, 22 (25%) with induction failure and 14 (16%) with relapsed disease. The median follow-up among survivors was 6.9 years (range 1.1-15.5). The 5- and 10-year PFS was 47% (95% CI: 36%-58%) and 38% (95% CI: 26%-50%), respectively. The 5- and 10-year OS was 53% (95% CI: 41%-63%) and 42% (95% CI: 29%-54%), respectively (Fig.1). At day 100 after allo-HCT, the rate of acute GVHD grade II-IV was 41% (95% CI: 30%-51). Chronic GVHD rates at 3 years were 62% (95% CI: 51%-72%), with extensive GVHD of 55% (95% CI: 44%-65%). On univariate analysis, age (> 60 or not), sex, TBI-based conditioning, donor type, stem cell source or remission status prior to allo-HCT did not predict for OS. Conclusions: This large single-institution series with a longterm follow-up on allo-HCT outcomes in patients with highrisk, aggressive T-cell NHL shows encouraging survival outcomes for these patients with limited treatment options.
Figure 1. Kaplan-Meier survival curves in patients with T-cell NHL undergoing allogeneic HCT.
POSTER SESSION I: MARROW FAILURE SYNDROMES (CONGENITAL AND ACQUIRED)
372 A Prospective Phase III Trial for Comparing Cyclophosphamide and Fludarabine Conditioning Regimen in Severe Idiopathic Aplastic Anemia Hawk Kim MD, PhD1, Eunji Choi MD2, Yunsuk Choi3, Won-Sik Lee4, Sung-Nam Lim5, Yong Park MD, PhD6, Hyeoung-Joon Kim MD, PhD7, Chul Won Jung MD8, Young Rok Do Professor9, Sukjoong Oh MD, PhD10, Jinny Park11, Je-Hwan Lee MD, PhD2, Kyoo-Hyung Lee MD, PhD2. 1 Division of Hematology, Gachon Univerisy Gil Medical Center, Gachon University College of Medicine, Incheon, Korea, Republic of (South); 2 Hematology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea, Republic of (South); 3 Division of Hematology and Hematological Malignancies, Ulsan University Hospital, University of Ulsan College of Medicine, Ulsan, Korea, Republic of (South); 4 Department of Internal Medicine, Inje University Busan Paik Hospital, Busan, Korea, Republic of (South); 5 Internal Medicine, Inje University College of Medicine, Heaundae Paik Hospital, Busan, Korea, Republic of (South); 6 Department of Medical Oncology and Hematology, Korea University Anam Hospital, Korea University College of Medicine, Seoul, Korea, Republic of (South); 7 Dept. of Hemato-Oncology, Chonnam National University Hospital, Kwangju, Korea (South); 8 Cancer Center, Samsung Medical Center, Seoul, Korea (South); 9 Hemato/Oncology, Keimyung University Dongsan Medical Center, Daegu, Korea, Republic of (South); 10 Department of Hematology and Oncology, Kangbuk Samsung Hospital, Sungkyunkwan University College of Medicine, Seoul, Korea, Republic of (South); 11 Division of Hematology, Gachon University Gil Medical Center, Gachon University College of Medcine, Incheon, Korea, Republic of (South) Our previous study showed that a less toxic regimen comprising reduced cyclophosphamide (Cy), fludarabine and anti-thymocyte globulin (ATG) (Cy-Flu-ATG), was less toxic for allogeneic hematopoietic cell transplantation (alloHCT) compared with standard Cy-ATG in patients with adult severe aplastic anemia (AA). We postulated that replacing Cy with Flu (Flu-ATG) would be more beneficial. Therefore, we performed a randomized phase III study to compare the regimen-related
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Abstracts / Biol Blood Marrow Transplant 26 (2020) S96 S255
toxicities (RRTs) of two different conditioning regimens: CyATG vs. Flu-ATG. Pre-defined RRTs were defined as pulmonary complications, hepatic sinusoidal obstruction syndrome (SOS), hemorrhagic cystitis and death of any causes. We present the final analysis. Patients in the Cy-ATG arm received Cy at 200 mg/kg. Those in the Flu-ATG arm received fludarabine (Flu) at 180 mg/m2. Bone marrow (BM) as allowed when the donor was matched sibling donor and otherwise stem cell source was mobilized peripheral blood (PB). Regimen for graft-versus-host disease (GvHD) was cyclosporine and shortcourse methotrexate. A total of 63 patients (31 patients in CyATG and 32 patients in Flu-ATG) were enrolled. The basic patients’characteristics were similar between both arms in terms of gender, age, prior immune suppression therapy history, stem cell source, stem cell dose, donor type and HLAmatching. There were 36 unrelated donors in each arm (p=1.000). All predefined RRTs were similar between Cy-ATG and Flu-ATG (33.3% vs. 21.9%; p = 0.691). There was no difference between Cy-ATG and Flu-ATG in terms of pulmonary complications (12.9% vs. 3.1%; p = 1.000), SOS (0% vs. 3.1%; p = 1.000), hemorrhagic cystitis (6.5% vs. 3.1%; p = 0.607) and death of any causes (23.3% vs. 15.6%; p = 0.443). Primary engraftment failure was not found in Flu-ATG arm but 2 patients in Cy-ATG (p = 0.230), one of which died of treatment-related hepatic toxicity before engraftment. However, there was higher secondary engraftment failure in Flu-ATG arms compared with Cy-ATG (2nd GF; 21.9% vs. 6.9%; p = 0.098). Therefore, any engraftment failure was not different between Cy-ATG vs. Flu-ATG (13.3% vs. 21.9%; p = 0.379). The incidence of all grades acute GvHD was significantly higher in Cy-ATG arm (35.7% vs. 9.4%; p = 0.013). The incidence of chronic GvHD was lower in Cy-ATG arm but was not statistically significant (11.5% vs. 23.1%; p = 0.271). Any significant treatment-related toxicities including RRTs were similar between Cy-ATG and Flu-ATG arms (43.3% vs. 43.8%; p = 0.974). The 3-year survival rate did not differ between Cy-ATG and Flu-ATG (72.9% vs. 79.3%; p = 0.329). In conclusion, Flu-ATG can be comparable with Cy-ATG in terms of RRT and survival.
373 Outcome of Aplastic Anemia Using Combined G-CSF Primed Blood and Bone Marrow Stem Cells a Retrospective Analysis Natasha Ali, Associate Professor Haematology1, Bakhtawer Altaf2, M Usman Shaikh Associate Professor Haematology3, Salman AdilProfessor Haematology4. 1 Pathology & Laboratory Medicine/Oncology, Aga Khan University Hospital, Karachi, Pakistan; 2 Oncology, Aga Khan University, Karachi, Pakistan; 3 The Aga Khan University, Karachi, Pakistan; 4 The Aga Khan University, Karachi, Pakistan Introduction: Aplastic anemia is characterized by diminished or absent hematopoietic precursors in the bone marrow, most often due to injury to the pluripotent stem cell. In Pakistan, Aplastic Anemia is not uncommon and allogeneic hematopoietic stem cell transplant remains the only curative option in these patients. Objective: To determine the transplant outcome of combined, G-CSF primed blood and bone marrow grafts in adult and pediatric patients with aplastic anemia. Methods: We retrospectively collected data of all transplant procedures performed from 2004-2018 at Aga Khan University Karachi, Pakistan. Variables analyzed included type of transplants, age, gender, indications for all transplant procedures and type of stem cells used, conditioning regimens and overall survival for patients undergoing transplant in aplastic anemia.
Results: A total of 314 transplants were performed during the study period. Out of these, 204 were allogeneic transplants whereas 110 were autologous procedures. There were 227 males and 87 females. The main indications for allogeneic stem cell transplant were aplastic anemia (60), acute leukemia (76) and b thalassemia major (39). Out of 60 patients with aplastic anemia, 44 were males and 16 were females. Fifty percent of patients were from pediatric age group (age >15 years). The median age § SD was 17.9 § 9.1 years (range: 2-43 years). Cyclophosphamide/ATG was used as a conditioning regimen in 56 patients, while ATG/cyclophosphamide/fludarabine was used in 2. Haploidentical transplant was done in 2 patients. Thirty seven percent patients underwent sex mismatched procedures. In 43 patients, a combination of GCSF primed blood and bone marrow stem cells were used. The mean CD34 count was 5.2 £ 106/kg. GVHD prophylaxis was done with cyclosporine and methotrexate. All patients received standard infection prophylaxis. Engraftment was achieved in 87% of patients. The median day of myeloid engraftment was 12 (range 9-15 days). Chronic GVHD was present in n=3 patients while n=4 had acute GVHD with an overall survival of 70% (median duration of 150 months). Conclusion: Combination of blood and bone marrow stem cells results in early engraftment with decreased frequency of GVHD in aplastic anemia. The overall survival was comparable to international literature.
374 First Line Haploidentical Stem Cell Transplantation in Children and Adolescents with Severe Aplastic Anemia Using Mobilized Peripheral Blood As Source of CD34+ Cells Maria Guadalupe Gonzalez Villarreal1, ~ o Luevano1, Myrna Patricia Pequen Severiano Baltazar Arellano2, Adriana Sandoval Gonzalez3, zquez4, Gerardo Martínez Pozos3, Andres Ortega Va Jose Alfredo Carrizales Villarreal3, Jose Luis Cedillo Cerda3, vila3, Alba Nydia Ramírez Lo pez3, Helga Patricia Sorkee Da Diego Gustavo Cruz Contreras3, Martha Lilia Guajardo Leal3, n Cantu 5, Guillermo Sotomayor Duque3, Rosa Elva De Leo ndez Valdez6. 1 Bone Marrow Transplantation, Roberto Herna Instituto Mexicano del Seguro Social, Monterrey, NL, Mexico; 2 Chief of Bone Marrow Transplantation, Instituto Mexicano del Seguro Social, Monterrey, NL, Mexico; 3 Instituto Mexicano del Seguro Social, Monterrey, NL, Mexico; 4 ISSSTE, Monterrey, NL, Mexico; 5 Chief of Hematology, Instituto Mexicano del Seguro Social, Monterrey, NL, Mexico; 6 Chief of Hemato-Oncology, Instituto Mexicano del Seguro Social, Monterrey, NL, Mexico Introduction: The only curative treatment for severe aplastic anemia in children is allogenic stem cell transplantation, but only few patients have a matched related or unrelated donor. Haploidentical stem cell transplantation (haplo-SCT) using bone marrow (BM) and peripheral blood stem cells (PBSC) has been recently described as effective and safe. In this study, we retrospectively describe the outcome of nine pediatric patients who underwent haplo-SCT using only PBSC. Patients and methods: The father was the selected donor for seven of the nine patients. Donor specific anti-HLA antibodies were ruled out. Stem cells mobilization was performed with filgrastim 10 mcg/kg/d for five days, and CD34+ were recollected by peripheral blood apheresis on day 0. The conditioning regimen consisted on rabbit anti-thymocyte globulin 2.5 mg/kg/d on days -7, -6, -5, and -4, and cyclophosphamide 50 mg/kg/d on days -3 and -2. We used cyclophosphamide 50 mg/kg/d on days +3 and +4, tacrolimus and mycophenolic acid as graft versus host disease prophylaxis. Results: The median follow up was 253 days (range 45-765 days). The median infused CD34+ was 10 £ 10^6/kg (range 2.7
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371 Long Term Outcomes of Patients with Aggressive T-Cell Non-Hodgkin Lymphoma Undergoing Allogeneic Stem Cell Transplantation: Retrospective Results from Single Center. Amandeep Salhotra MD1, Liana Nikolaenko MD2, Lu Chen PhD3, Ni-Chun Tsai MS, MPH4, D. Lynne Smith PhD2, Auayporn Nademanee MD5, Leslie Popplewell MD, FACP5, Alex F. Herrera MD2, Matthew Mei MD5, Stephen J. Forman MD5, Jasmine Zain MD5. 1 Hematology/Hematopoietic Cell Transplantation, City of Hope National Medical Center, Duarte, CA; 2 Hematology and Hematopoietic Cell Transplantation, City of Hope National Medical Center, Duarte, CA; 3 Computational and Quantitative Medicine, City of Hope National Medical Center, Duarte, CA; 4 Computational and Quantitative Medicine-BRI, City of Hope National Medical Center, Duarte, CA; 5 Hematology/ Hematopoietic Cell Transplant, City of Hope National Medical Center, Duarte, CA Introduction: Peripheral T-cell lymphomas (PTCL) comprise 1520% of adult non-Hodgkin lymphoma and have a poor prognosis; 5-year survival is less than 30% for the most aggressive subtypes. Allogeneic HCT (allo-HCT) is offered to eligible patients as a potentially curative modality in the salvage setting or in high risk patients to consolidate an initial response to frontline therapy. Objective: To report clinical outcomes derived from large sample size and long-term follow up data. Methods: We retrospectively reviewed medical records of 87 consecutive patients with PTCL who underwent allo-HCT at City of Hope from January 2000 to June 2018. Baseline patient demographic, treatment, and disease characteristics were summarized by descriptive statistics. Overall survival (OS) and progressionfree survival (PFS) were evaluated by Kaplan-Meier curves and the log-rank test. Cumulative incidences of time to relapse and time to non-relapse mortality (NRM) were calculated with relapse and NRM as competing risks. Cumulative incidences of acute and chronic GVHD were calculated as time to onset of GVHD with relapse and death as competing events for GVHD. Results: Median age at allo-HCT was 49 (range 2-70) years. Histologies were PTCL-NOS (n=21); transformed CTCL (n=19); NK TCL (n=17); AITL (n=15), ALCL (n=7); gdTCL (n=6) and other rare subtypes (n=2). No patients had a prior auto transplant. 42 patients (48%) had myeloablative conditioning; FTBI-based (n=39), or BEAM regimen (n=3). 45 patients (52%) had reduced intensity conditioning with a fludarabine/melphalan based regimen in 39. Sibling HCT was done in 47 (54%) patients. MUD HCT was done in 36 (41%); donors were fully HLA matched for 15 (17%) patients and mismatched in 21 (24%); 4 (5%) got haploidentical HCT. The most common GVHD prophylaxis was tacrolimus/sirolimus (n=54). Stem cell source was PBSC in 77 (88%), bone marrow in 5 (6%), and cord blood in 5 (6%) patients. At allo-HCT 25 (29%) patients were in complete remission, 25 (29%) in partial remission, 22 (25%) with induction failure and 14 (16%) with relapsed disease. The median follow-up among survivors was 6.9 years (range 1.1-15.5). The 5- and 10-year PFS was 47% (95% CI: 36%-58%) and 38% (95% CI: 26%-50%), respectively. The 5- and 10-year OS was 53% (95% CI: 41%-63%) and 42% (95% CI: 29%-54%), respectively (Fig.1). At day 100 after allo-HCT, the rate of acute GVHD grade II-IV was 41% (95% CI: 30%-51). Chronic GVHD rates at 3 years were 62% (95% CI: 51%-72%), with extensive GVHD of 55% (95% CI: 44%-65%). On univariate analysis, age (> 60 or not), sex, TBI-based conditioning, donor type, stem cell source or remission status prior to allo-HCT did not predict for OS. Conclusions: This large single-institution series with a longterm follow-up on allo-HCT outcomes in patients with highrisk, aggressive T-cell NHL shows encouraging survival outcomes for these patients with limited treatment options.
Figure 1. Kaplan-Meier survival curves in patients with T-cell NHL undergoing allogeneic HCT.
POSTER SESSION I: MARROW FAILURE SYNDROMES (CONGENITAL AND ACQUIRED)
372 A Prospective Phase III Trial for Comparing Cyclophosphamide and Fludarabine Conditioning Regimen in Severe Idiopathic Aplastic Anemia Hawk Kim MD, PhD1, Eunji Choi MD2, Yunsuk Choi3, Won-Sik Lee4, Sung-Nam Lim5, Yong Park MD, PhD6, Hyeoung-Joon Kim MD, PhD7, Chul Won Jung MD8, Young Rok Do Professor9, Sukjoong Oh MD, PhD10, Jinny Park11, Je-Hwan Lee MD, PhD2, Kyoo-Hyung Lee MD, PhD2. 1 Division of Hematology, Gachon Univerisy Gil Medical Center, Gachon University College of Medicine, Incheon, Korea, Republic of (South); 2 Hematology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea, Republic of (South); 3 Division of Hematology and Hematological Malignancies, Ulsan University Hospital, University of Ulsan College of Medicine, Ulsan, Korea, Republic of (South); 4 Department of Internal Medicine, Inje University Busan Paik Hospital, Busan, Korea, Republic of (South); 5 Internal Medicine, Inje University College of Medicine, Heaundae Paik Hospital, Busan, Korea, Republic of (South); 6 Department of Medical Oncology and Hematology, Korea University Anam Hospital, Korea University College of Medicine, Seoul, Korea, Republic of (South); 7 Dept. of Hemato-Oncology, Chonnam National University Hospital, Kwangju, Korea (South); 8 Cancer Center, Samsung Medical Center, Seoul, Korea (South); 9 Hemato/Oncology, Keimyung University Dongsan Medical Center, Daegu, Korea, Republic of (South); 10 Department of Hematology and Oncology, Kangbuk Samsung Hospital, Sungkyunkwan University College of Medicine, Seoul, Korea, Republic of (South); 11 Division of Hematology, Gachon University Gil Medical Center, Gachon University College of Medcine, Incheon, Korea, Republic of (South) Our previous study showed that a less toxic regimen comprising reduced cyclophosphamide (Cy), fludarabine and anti-thymocyte globulin (ATG) (Cy-Flu-ATG), was less toxic for allogeneic hematopoietic cell transplantation (alloHCT) compared with standard Cy-ATG in patients with adult severe aplastic anemia (AA). We postulated that replacing Cy with Flu (Flu-ATG) would be more beneficial. Therefore, we performed a randomized phase III study to compare the regimen-related
S250
Abstracts / Biol Blood Marrow Transplant 26 (2020) S96 S255
toxicities (RRTs) of two different conditioning regimens: CyATG vs. Flu-ATG. Pre-defined RRTs were defined as pulmonary complications, hepatic sinusoidal obstruction syndrome (SOS), hemorrhagic cystitis and death of any causes. We present the final analysis. Patients in the Cy-ATG arm received Cy at 200 mg/kg. Those in the Flu-ATG arm received fludarabine (Flu) at 180 mg/m2. Bone marrow (BM) as allowed when the donor was matched sibling donor and otherwise stem cell source was mobilized peripheral blood (PB). Regimen for graft-versus-host disease (GvHD) was cyclosporine and shortcourse methotrexate. A total of 63 patients (31 patients in CyATG and 32 patients in Flu-ATG) were enrolled. The basic patients’characteristics were similar between both arms in terms of gender, age, prior immune suppression therapy history, stem cell source, stem cell dose, donor type and HLAmatching. There were 36 unrelated donors in each arm (p=1.000). All predefined RRTs were similar between Cy-ATG and Flu-ATG (33.3% vs. 21.9%; p = 0.691). There was no difference between Cy-ATG and Flu-ATG in terms of pulmonary complications (12.9% vs. 3.1%; p = 1.000), SOS (0% vs. 3.1%; p = 1.000), hemorrhagic cystitis (6.5% vs. 3.1%; p = 0.607) and death of any causes (23.3% vs. 15.6%; p = 0.443). Primary engraftment failure was not found in Flu-ATG arm but 2 patients in Cy-ATG (p = 0.230), one of which died of treatment-related hepatic toxicity before engraftment. However, there was higher secondary engraftment failure in Flu-ATG arms compared with Cy-ATG (2nd GF; 21.9% vs. 6.9%; p = 0.098). Therefore, any engraftment failure was not different between Cy-ATG vs. Flu-ATG (13.3% vs. 21.9%; p = 0.379). The incidence of all grades acute GvHD was significantly higher in Cy-ATG arm (35.7% vs. 9.4%; p = 0.013). The incidence of chronic GvHD was lower in Cy-ATG arm but was not statistically significant (11.5% vs. 23.1%; p = 0.271). Any significant treatment-related toxicities including RRTs were similar between Cy-ATG and Flu-ATG arms (43.3% vs. 43.8%; p = 0.974). The 3-year survival rate did not differ between Cy-ATG and Flu-ATG (72.9% vs. 79.3%; p = 0.329). In conclusion, Flu-ATG can be comparable with Cy-ATG in terms of RRT and survival.
373 Outcome of Aplastic Anemia Using Combined G-CSF Primed Blood and Bone Marrow Stem Cells a Retrospective Analysis Natasha Ali, Associate Professor Haematology1, Bakhtawer Altaf2, M Usman Shaikh Associate Professor Haematology3, Salman AdilProfessor Haematology4. 1 Pathology & Laboratory Medicine/Oncology, Aga Khan University Hospital, Karachi, Pakistan; 2 Oncology, Aga Khan University, Karachi, Pakistan; 3 The Aga Khan University, Karachi, Pakistan; 4 The Aga Khan University, Karachi, Pakistan Introduction: Aplastic anemia is characterized by diminished or absent hematopoietic precursors in the bone marrow, most often due to injury to the pluripotent stem cell. In Pakistan, Aplastic Anemia is not uncommon and allogeneic hematopoietic stem cell transplant remains the only curative option in these patients. Objective: To determine the transplant outcome of combined, G-CSF primed blood and bone marrow grafts in adult and pediatric patients with aplastic anemia. Methods: We retrospectively collected data of all transplant procedures performed from 2004-2018 at Aga Khan University Karachi, Pakistan. Variables analyzed included type of transplants, age, gender, indications for all transplant procedures and type of stem cells used, conditioning regimens and overall survival for patients undergoing transplant in aplastic anemia.
Results: A total of 314 transplants were performed during the study period. Out of these, 204 were allogeneic transplants whereas 110 were autologous procedures. There were 227 males and 87 females. The main indications for allogeneic stem cell transplant were aplastic anemia (60), acute leukemia (76) and b thalassemia major (39). Out of 60 patients with aplastic anemia, 44 were males and 16 were females. Fifty percent of patients were from pediatric age group (age >15 years). The median age § SD was 17.9 § 9.1 years (range: 2-43 years). Cyclophosphamide/ATG was used as a conditioning regimen in 56 patients, while ATG/cyclophosphamide/fludarabine was used in 2. Haploidentical transplant was done in 2 patients. Thirty seven percent patients underwent sex mismatched procedures. In 43 patients, a combination of GCSF primed blood and bone marrow stem cells were used. The mean CD34 count was 5.2 £ 106/kg. GVHD prophylaxis was done with cyclosporine and methotrexate. All patients received standard infection prophylaxis. Engraftment was achieved in 87% of patients. The median day of myeloid engraftment was 12 (range 9-15 days). Chronic GVHD was present in n=3 patients while n=4 had acute GVHD with an overall survival of 70% (median duration of 150 months). Conclusion: Combination of blood and bone marrow stem cells results in early engraftment with decreased frequency of GVHD in aplastic anemia. The overall survival was comparable to international literature.
374 First Line Haploidentical Stem Cell Transplantation in Children and Adolescents with Severe Aplastic Anemia Using Mobilized Peripheral Blood As Source of CD34+ Cells Maria Guadalupe Gonzalez Villarreal1, ~ o Luevano1, Myrna Patricia Pequen Severiano Baltazar Arellano2, Adriana Sandoval Gonzalez3, zquez4, Gerardo Martínez Pozos3, Andres Ortega Va Jose Alfredo Carrizales Villarreal3, Jose Luis Cedillo Cerda3, vila3, Alba Nydia Ramírez Lo pez3, Helga Patricia Sorkee Da Diego Gustavo Cruz Contreras3, Martha Lilia Guajardo Leal3, n Cantu 5, Guillermo Sotomayor Duque3, Rosa Elva De Leo ndez Valdez6. 1 Bone Marrow Transplantation, Roberto Herna Instituto Mexicano del Seguro Social, Monterrey, NL, Mexico; 2 Chief of Bone Marrow Transplantation, Instituto Mexicano del Seguro Social, Monterrey, NL, Mexico; 3 Instituto Mexicano del Seguro Social, Monterrey, NL, Mexico; 4 ISSSTE, Monterrey, NL, Mexico; 5 Chief of Hematology, Instituto Mexicano del Seguro Social, Monterrey, NL, Mexico; 6 Chief of Hemato-Oncology, Instituto Mexicano del Seguro Social, Monterrey, NL, Mexico Introduction: The only curative treatment for severe aplastic anemia in children is allogenic stem cell transplantation, but only few patients have a matched related or unrelated donor. Haploidentical stem cell transplantation (haplo-SCT) using bone marrow (BM) and peripheral blood stem cells (PBSC) has been recently described as effective and safe. In this study, we retrospectively describe the outcome of nine pediatric patients who underwent haplo-SCT using only PBSC. Patients and methods: The father was the selected donor for seven of the nine patients. Donor specific anti-HLA antibodies were ruled out. Stem cells mobilization was performed with filgrastim 10 mcg/kg/d for five days, and CD34+ were recollected by peripheral blood apheresis on day 0. The conditioning regimen consisted on rabbit anti-thymocyte globulin 2.5 mg/kg/d on days -7, -6, -5, and -4, and cyclophosphamide 50 mg/kg/d on days -3 and -2. We used cyclophosphamide 50 mg/kg/d on days +3 and +4, tacrolimus and mycophenolic acid as graft versus host disease prophylaxis. Results: The median follow up was 253 days (range 45-765 days). The median infused CD34+ was 10 £ 10^6/kg (range 2.7