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Comparison of Peripheral Blood Versus Bone Marrow As Stem Cell Source from Matched-Related Donors on the Outcome of Allogeneic Hematopoietic Stem Cell Transplantation for Children with Acute Leukemia
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Abstracts / Biol Blood Marrow Transplant 22 (2016) S19eS481
survival such that for every 1 x10 6/kg unit increase there was a 1.8% increased risk of death, HR 1.018 (1.001-1.036). Additional information is needed to help guide practice and has implications for choosing the target cell dose prior to a bone marrow harvest and the management of bone marrow donors.
follow up is 39 months (range 2-148). The 5-year probability of overall and leukemia-free survival for those receiving PB compared to BM was (61% versus 52%, p¼0.416) and (66% versus 43% p¼0.022) respectively. Conclusion: The use of PB compared to BM as a stem cell source from pediatric donors for children with acute leukemia is feasible and resulted in faster platelets recovery, significantly less relapse and improved leukemia-free survival.
351 Comparison of Peripheral Blood Versus Bone Marrow As Stem Cell Source from Matched-Related Donors on the Outcome of Allogeneic Hematopoietic Stem Cell Transplantation for Children with Acute Leukemia Ayad Ahmed Hussein 1, Rula Najjar2, Khadra Salami 3, Anas Haroun Haroun3, Rand Farraj3, Abdulhadi I. Al-Zaben 4, Haydar A. Frangoul 5. 1 Bone Marrow and Stem Cell Transplantation Program, King Hussein Cancer Center, Amman, Jordan; 2 king hussein cancer center, amman, Jordan; 3 King Hussein Cancer Center (KHCC), Amman, Jordan; 4 Bone Marrow and Stem Cell Transplantation Program, King Hussein Cancer Center, amman, Jordan; 5 The Children’s Hospital at TriStar Centennial and Sarah Cannon Reseach Institute, Nashville, TN Introduction: The effect of stem cell source on the outcome of children with acute leukemia following hematopoietic stem cell transplantation (HSCT) from fully matched related donor in countries with high rate of consanguinity and high incidence of CMV exposure, is not well known. Patients and Methods: We retrospectively reviewed the medical records of all children with acute leukemia who received matched-related HSCT at the Bone Marrow and Stem Cell Transplantation program at King Hussein Cancer Center (KHCC) in Amman, Jordan from January 2003 to December 2014. Results: A total of 111 patients were included, with a median age of 10 years (1-20) and 65 (58.5%) were males. Fifty-seven patients had ALL (51%) and 54 had AML (49%). Patients with ALL and those with AML beyond CR1 received TBI (1200 cGy)/Cy (120 mg/kg) conditioning. Patients with AML in CR1 received Bu (16 mg/kg)/Cy (200 mg/kg) conditioning. Ninety-three patients (83%) received peripheral blood (PB) and 18 patients (16.2%) received bone marrow (BM) as stem cell source. Patients who received PB and BM had similar age (p¼.714), sex (p¼0.115), CR status (p¼0.079), donor age (¼0.216), patient-donor sex-match (p¼0.215), and patientdonor CMV status matching (p¼0.999). There were more patients with ALL (p¼0.003) in the PBSC group. The median age of donors was 11 years (1-20) for PB group and 5 years (2 -23) for the BM group (p¼0.216). The median time to neutrophil engraftment for the PB and BM groups was 14 (10-23) and 13 days (11-22) , respectively (p¼ 0.861). The median time to platelet engraftment for the PB and BM groups was 14 (10-29) and 17 days (10-40), respectively (p¼ 0.006). The median hospitalization after stem cell infusion was 20 days (13-133) for the PB group and 19 days (17-35) for the BM group (p¼0.187). The incidence of CMV reactivation in the first 100 days following HSCT was 44% for PB group and 33% for the BM group (p¼0.398), and no CMV disease reported in both groups. The incidence of acute grade II-III GVHD was 35% for the PB group and 33% for the BM group (p¼0.9). There was a trend for higher incidence of chronic GVHD among the PB group compared to the BM group (35% versus 11% respectively p¼0.052). The transplant-related mortality (TRM) was 3% for patients who received PB and 0% for those who received BM (p¼0.999). There was significantly higher relapse rate among those who received BM compared to PB (55% versus 30% respectively p¼0.0372). The median
352 Allogeneic Hematopoietic Stem Cell Transplantation Using a Reduced Intensity Conditioning Regimen for Adolescents and Young Adults with Class 3 Beta-Thalassemia Ayad Ahmed Hussein 1, Abdulhadi I. Al-Zaben 2, Eman Khattab 3, Anas Haroun 1, Haydar A. Frangoul 4. 1 Bone Marrow and Stem Cell Transplantation Program, King Hussein Cancer Center, Amman, Jordan; 2 Bone Marrow and Stem Cell Transplantation Program, King Hussein Cancer Center, amman, Jordan; 3 King Hussein Cancer Center, Amman, Jordan; 4 The Children’s Hospital at TriStar Centennial and Sarah Cannon Reseach Institute, Nashville, TN Introduction: Although allogeneic hematopoietic stem cell transplantat (HSCT) can offer a curative approach for patients with thalassemia major, the reported outcome for adolescents and young adults (AYA) is inferior to that in children. Patients and Methods: We report on all consecutive patients 14 year of age with thalassemia major who received allogeneic HSCT from matched family donors at the Bone Marrow and Stem Cell Transplantation program at King Hussein Cancer Center (KHCC) in Amman, Jordan from January, 2004 until February, 2015. Results: Twenty-nine patients were included, with a median age of 17 year (range 14-28). The median ferritin level was 2980 ng/dl (270-10850). All patients had liver biopsy that showed evidence of liver fibrosis and were classified as Pesaro Class 3. Eleven patients (37%) had hepatitis B or C infection. Twelve patients (41%) underwent splenectomy prior to HSCT. All patients received reduced intensity conditioning (RIC) consisted of oral busulfan 2 mg/kg given every 12 hours on days -8 to -7, fludarabine 35 mg/m2 on days -6 to -2, ATG (horse 30 mg/kg for on days -6 to -2, or Rabitt 2.5 mg/kg on days -3 to -1) and total lymphoid irradiation (TLI) administered as a single fraction of 500 cGy on day zero prior to stem cell infusion. Cyclosporine and mycophenolate were used for GVHD prophylaxis. Peripheral blood stem cells from fully matched related donors was used in all patients. All patients achieved neutrophil and platelet engraftment at a median time of 14 (10-24) and 16 days (11-28), respectively. Three patients (10%) developed grade II skin acute GVHD. Chronic GVHD developed in 5 patients (17%), all were controlled with first line treatment. Secondary graft failure was observed in 3 (10%) patients, all of them are transfusion independent following a second RIC HSCT using the same preparative regimen without TLI. There was no transplant related mortality. At a median follow up of 28 months (6-130), all patients are alive and transfusion independent. The 3-year probability of overall and thalassemia-free survival was 100% and 90%, respectively. Conclusion: Using a TLI based RIC in AYA patients with high risk thalassemia major is very well tolerated and can result in an excellent thalassemia free survival.
Abstracts / Biol Blood Marrow Transplant 22 (2016) S19eS481
survival such that for every 1 x10 6/kg unit increase there was a 1.8% increased risk of death, HR 1.018 (1.001-1.036). Additional information is needed to help guide practice and has implications for choosing the target cell dose prior to a bone marrow harvest and the management of bone marrow donors.
follow up is 39 months (range 2-148). The 5-year probability of overall and leukemia-free survival for those receiving PB compared to BM was (61% versus 52%, p¼0.416) and (66% versus 43% p¼0.022) respectively. Conclusion: The use of PB compared to BM as a stem cell source from pediatric donors for children with acute leukemia is feasible and resulted in faster platelets recovery, significantly less relapse and improved leukemia-free survival.
351 Comparison of Peripheral Blood Versus Bone Marrow As Stem Cell Source from Matched-Related Donors on the Outcome of Allogeneic Hematopoietic Stem Cell Transplantation for Children with Acute Leukemia Ayad Ahmed Hussein 1, Rula Najjar2, Khadra Salami 3, Anas Haroun Haroun3, Rand Farraj3, Abdulhadi I. Al-Zaben 4, Haydar A. Frangoul 5. 1 Bone Marrow and Stem Cell Transplantation Program, King Hussein Cancer Center, Amman, Jordan; 2 king hussein cancer center, amman, Jordan; 3 King Hussein Cancer Center (KHCC), Amman, Jordan; 4 Bone Marrow and Stem Cell Transplantation Program, King Hussein Cancer Center, amman, Jordan; 5 The Children’s Hospital at TriStar Centennial and Sarah Cannon Reseach Institute, Nashville, TN Introduction: The effect of stem cell source on the outcome of children with acute leukemia following hematopoietic stem cell transplantation (HSCT) from fully matched related donor in countries with high rate of consanguinity and high incidence of CMV exposure, is not well known. Patients and Methods: We retrospectively reviewed the medical records of all children with acute leukemia who received matched-related HSCT at the Bone Marrow and Stem Cell Transplantation program at King Hussein Cancer Center (KHCC) in Amman, Jordan from January 2003 to December 2014. Results: A total of 111 patients were included, with a median age of 10 years (1-20) and 65 (58.5%) were males. Fifty-seven patients had ALL (51%) and 54 had AML (49%). Patients with ALL and those with AML beyond CR1 received TBI (1200 cGy)/Cy (120 mg/kg) conditioning. Patients with AML in CR1 received Bu (16 mg/kg)/Cy (200 mg/kg) conditioning. Ninety-three patients (83%) received peripheral blood (PB) and 18 patients (16.2%) received bone marrow (BM) as stem cell source. Patients who received PB and BM had similar age (p¼.714), sex (p¼0.115), CR status (p¼0.079), donor age (¼0.216), patient-donor sex-match (p¼0.215), and patientdonor CMV status matching (p¼0.999). There were more patients with ALL (p¼0.003) in the PBSC group. The median age of donors was 11 years (1-20) for PB group and 5 years (2 -23) for the BM group (p¼0.216). The median time to neutrophil engraftment for the PB and BM groups was 14 (10-23) and 13 days (11-22) , respectively (p¼ 0.861). The median time to platelet engraftment for the PB and BM groups was 14 (10-29) and 17 days (10-40), respectively (p¼ 0.006). The median hospitalization after stem cell infusion was 20 days (13-133) for the PB group and 19 days (17-35) for the BM group (p¼0.187). The incidence of CMV reactivation in the first 100 days following HSCT was 44% for PB group and 33% for the BM group (p¼0.398), and no CMV disease reported in both groups. The incidence of acute grade II-III GVHD was 35% for the PB group and 33% for the BM group (p¼0.9). There was a trend for higher incidence of chronic GVHD among the PB group compared to the BM group (35% versus 11% respectively p¼0.052). The transplant-related mortality (TRM) was 3% for patients who received PB and 0% for those who received BM (p¼0.999). There was significantly higher relapse rate among those who received BM compared to PB (55% versus 30% respectively p¼0.0372). The median
352 Allogeneic Hematopoietic Stem Cell Transplantation Using a Reduced Intensity Conditioning Regimen for Adolescents and Young Adults with Class 3 Beta-Thalassemia Ayad Ahmed Hussein 1, Abdulhadi I. Al-Zaben 2, Eman Khattab 3, Anas Haroun 1, Haydar A. Frangoul 4. 1 Bone Marrow and Stem Cell Transplantation Program, King Hussein Cancer Center, Amman, Jordan; 2 Bone Marrow and Stem Cell Transplantation Program, King Hussein Cancer Center, amman, Jordan; 3 King Hussein Cancer Center, Amman, Jordan; 4 The Children’s Hospital at TriStar Centennial and Sarah Cannon Reseach Institute, Nashville, TN Introduction: Although allogeneic hematopoietic stem cell transplantat (HSCT) can offer a curative approach for patients with thalassemia major, the reported outcome for adolescents and young adults (AYA) is inferior to that in children. Patients and Methods: We report on all consecutive patients 14 year of age with thalassemia major who received allogeneic HSCT from matched family donors at the Bone Marrow and Stem Cell Transplantation program at King Hussein Cancer Center (KHCC) in Amman, Jordan from January, 2004 until February, 2015. Results: Twenty-nine patients were included, with a median age of 17 year (range 14-28). The median ferritin level was 2980 ng/dl (270-10850). All patients had liver biopsy that showed evidence of liver fibrosis and were classified as Pesaro Class 3. Eleven patients (37%) had hepatitis B or C infection. Twelve patients (41%) underwent splenectomy prior to HSCT. All patients received reduced intensity conditioning (RIC) consisted of oral busulfan 2 mg/kg given every 12 hours on days -8 to -7, fludarabine 35 mg/m2 on days -6 to -2, ATG (horse 30 mg/kg for on days -6 to -2, or Rabitt 2.5 mg/kg on days -3 to -1) and total lymphoid irradiation (TLI) administered as a single fraction of 500 cGy on day zero prior to stem cell infusion. Cyclosporine and mycophenolate were used for GVHD prophylaxis. Peripheral blood stem cells from fully matched related donors was used in all patients. All patients achieved neutrophil and platelet engraftment at a median time of 14 (10-24) and 16 days (11-28), respectively. Three patients (10%) developed grade II skin acute GVHD. Chronic GVHD developed in 5 patients (17%), all were controlled with first line treatment. Secondary graft failure was observed in 3 (10%) patients, all of them are transfusion independent following a second RIC HSCT using the same preparative regimen without TLI. There was no transplant related mortality. At a median follow up of 28 months (6-130), all patients are alive and transfusion independent. The 3-year probability of overall and thalassemia-free survival was 100% and 90%, respectively. Conclusion: Using a TLI based RIC in AYA patients with high risk thalassemia major is very well tolerated and can result in an excellent thalassemia free survival.