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Transplantation Of Haploidentical Family Donor Stem Cells Combining With Cord Blood Transfusion For Treatment Of Hematologic Malignancies In Pediatric Patients

Posted on:2015-11-02Degree:MasterType:Thesis
Country:ChinaCandidate:Y LuFull Text:PDF
GTID:2284330431451602Subject:Academy of Pediatrics
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Objective:Allogeneic hematopoietic stem cell transplantation (Allo HSCT) is themain method of the treatment that can cure hematologic malignancies for children. Withrecent advances in transplant technology, haploidentical hematopoietic stem cells (HaploSCT) as an important source of stem cells has been widely used. In this study, weretrospectively analyze the efficacy and safety of Haplo SCT combining with cord blood(CB) transfusion for children with hematologic malignancies in our medical center.Methods:1, Patient characteristics: Six pediatric patients were enrolled from August untilNovember2012, including2cases of acute lymphocytic leukemia (ALL),2cases of acutemyelocytic leukemia (AML),1case of chronic myelogenous leukemia (CML), and1acase of severe aplastic anemia (SAA). Median age was10years. Median weight was38.3Kg. G-CSF–mobilized bone marrow (BM) and peripheral blood (PB) stem cells wereused as a graft resource, without in vitro T-cell depletion. The median number ofmononuclear cell dose was7.00(range,1.51to12.13)×108/Kg, and CD34+cell dose was1.81(range,1.23to2.45)×106/Kg coming from bone marrow. In the peripheral blood, themedian number of mononuclear cell dose and CD34+cell dose were7.96(range,4.09to14.64)×108/Kg and5.85(range,2.74to14.20)×106/Kg, respectively. Cord blood wasinfused four hours earlier than bone marrow stem cells and one more day than peripheralblood stem cells. The individualized and myeloablative conditioning regimen was amodification of BU/CY or CY/TBI. After transplantation, complete blood count (CBC) was daily monitored. All transplantation recipients received cyclosporine A (CsA),anti-human thymocyte globulin (ATG), mycophenolate mofetil (MMF), and short-termmethotrexate (MTX) as graft-versus-host disease (GVHD) prophylaxis. Othercomplications were be prevented and treated actively. All patients were followed sinceHaplo SCT.2, Detection before transplantation: Donors and recipients were detected humanleukocyte antigen (HLA), HLA antibodies, major histocompatibility complex class Ichain-related gene A (MICA) antibodies and killer cell Ig-like receptor (KIR). In order toimprove the success rate of transplants, rituximab was given to recipients beforetransplantation.3, Chimerism was determined by short tandem repeat(STR). It can be use to predictthe risk of recurrence.4, Detection of post-transplant: Recipients were monitored cytomegalovirus-PP65(CMV-PP65), CMV-DNA and BK polyomavirus (BKV). Prevention and earlyidentification were the key point to reduce the occurrence and severity of complicationsafter transplantation.Results:1, One patient who had HLA-antibodies received rituximab before transplantation.MICA antibodies were negative in all patients. Two cases were KIR-mismatched.2, Six patients achieved sustained donor engraftment. The median time of neutrophilengraftment was11.8(range,10to15) days. The median time of platelet engraftment was14.2(range,12to16) days. The median time of STR≥95%which was considered ascomplete engraftment was18(range,14to21) days.3, Two patients occurred degree II of acute graft-versus-host disease (aGVHD), nochronic graft-versus-host disease(cGVHD). Hemorrhagic cystitis (HC) developed in twopatients, and one of them had BKV infection. Of the six patients, five patients had CMVinfection, and three of them had infected recurrently. One case was diagnosed as invasivefungal pneumonia (IFP). No case had veno occlusive disease (VOD). 4, The median follow-up was376.5(range,136to496) days,5patients weredisease-free survival, only one case with SAA died of interstitial pneumonia on day136.Conclusion: Haplo SCT combining with CB transfusion results in rapid engraftment,low GVHD, and high proportion of disease-free survival.
Keywords/Search Tags:haploidentical hematopoietic stem cells (Haplo SCT), cord blood (CB), stem cell transplantation (SCT), engraftment, graft-versus-host disease(GVHD), children
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