{"eventGroups":[{"description":"All eligible patients received pre-transplant conditioning with Alemtuzumab, fludarabine and targeted busulfan followed by allogeneic transplant. The initial dose of busulfan was determined by performing PK (pharmacokinetics) analysis on a test dose of busulfan on the day prior to initiating conditioning. Based on the PK results a starting dose of busulfan was determined. A second PK study was done after the starting dose to ensure that the targeted dose was achieved. If it wasn't achieved, then a dose modification was made and a third PK study done.","id":"EG000","otherNumAffected":33,"otherNumAtRisk":35,"seriousNumAffected":7,"seriousNumAtRisk":35,"title":"Single Arm - Transplant Pre-conditioning Per Study Protocol"}],"frequencyThreshold":"5","otherEvents":[{"assessmentType":"SYSTEMATIC_ASSESSMENT","notes":"\\< Grade III - 17 participants; Grade III - 15 participants; Grade IV - 1 participant","organSystem":"Gastrointestinal disorders","stats":[{"groupId":"EG000","numAffected":33,"numAtRisk":35,"numEvents":33}],"term":"Mucositis (All grades)"},{"assessmentType":"SYSTEMATIC_ASSESSMENT","notes":"aGVHD \\< Grade IV","organSystem":"General disorders","stats":[{"groupId":"EG000","numAffected":5,"numAtRisk":35,"numEvents":5}],"term":"aGVHD"},{"assessmentType":"SYSTEMATIC_ASSESSMENT","organSystem":"General disorders","stats":[{"groupId":"EG000","numAffected":2,"numAtRisk":35,"numEvents":2}],"term":"cGVHD (Chronic graft-versus-host disease)"},{"assessmentType":"SYSTEMATIC_ASSESSMENT","organSystem":"General disorders","stats":[{"groupId":"EG000","numAffected":19,"numAtRisk":35,"numEvents":19}],"term":"Infusion reaction to alemtuzumab"}],"seriousEvents":[{"assessmentType":"SYSTEMATIC_ASSESSMENT","notes":"acute Graft-Versus-Host Disease","organSystem":"General disorders","stats":[{"groupId":"EG000","numAffected":1,"numAtRisk":35,"numEvents":1}],"term":"aGVHD (acute Graft-Versus-Host Disease)"},{"assessmentType":"NON_SYSTEMATIC_ASSESSMENT","notes":"After 4th unrelated donor transplant (off study), patient developed respiratory distress that was associated with a CMV infection. Respiratory distress worsened in spite of maximal therapy. The parents elected to withdraw therapy and he died.","organSystem":"Respiratory, thoracic and mediastinal disorders","stats":[{"groupId":"EG000","numAffected":1,"numAtRisk":35,"numEvents":1}],"term":"Respiratory distress associated with Cytomegalovirus infection"},{"assessmentType":"NON_SYSTEMATIC_ASSESSMENT","notes":"Child presented with fever and pancytopenia, splenomegaly and was admitted for possible recurrent disease.","organSystem":"Blood and lymphatic system disorders","stats":[{"groupId":"EG000","numAffected":1,"numAtRisk":35,"numEvents":1}],"term":"Pancytopenia and splenomegaly"},{"assessmentType":"NON_SYSTEMATIC_ASSESSMENT","notes":"Worsening respiratory status despite maximal support, at parent's request, patient was removed from mechanical ventilation, and the patient died quickly and peacefully.","organSystem":"Respiratory, thoracic and mediastinal disorders","stats":[{"groupId":"EG000","numAffected":1,"numAtRisk":35,"numEvents":1}],"term":"Repiratory failure following ideopathic pneumonia syndrome"},{"assessmentType":"NON_SYSTEMATIC_ASSESSMENT","notes":"Sudden onset of thrombocytopenia of unknown etiology.","organSystem":"Blood and lymphatic system disorders","stats":[{"groupId":"EG000","numAffected":1,"numAtRisk":35,"numEvents":1}],"term":"Thrombocytopenia"},{"assessmentType":"NON_SYSTEMATIC_ASSESSMENT","notes":"Patient admitted with cough and fever and diagnosed with Aspergillus pneumonia.","organSystem":"Infections and infestations","stats":[{"groupId":"EG000","numAffected":1,"numAtRisk":35,"numEvents":1}],"term":"Aspergillus pneumonia"},{"assessmentType":"NON_SYSTEMATIC_ASSESSMENT","notes":"Patient developed renal failure and is required dialysis","organSystem":"Renal and urinary disorders","stats":[{"groupId":"EG000","numAffected":1,"numAtRisk":35,"numEvents":1}],"term":"Renal Failure"},{"assessmentType":"SYSTEMATIC_ASSESSMENT","notes":"initially, cells recovered normally with neutrophil count \\>500 by day +12 post-transplant. By day +20 they began a slow decline and by day +40 the neutrophil count was \\<500 with 99% host CD3+ (cluster of differentiation 3-positive) cells.","organSystem":"Blood and lymphatic system disorders","stats":[{"groupId":"EG000","numAffected":1,"numAtRisk":35,"numEvents":1}],"term":"Graft rejection/failure"},{"assessmentType":"NON_SYSTEMATIC_ASSESSMENT","organSystem":"Infections and infestations","stats":[{"groupId":"EG000","numAffected":1,"numAtRisk":35,"numEvents":1}],"term":"Viral Encephalitis"},{"assessmentType":"NON_SYSTEMATIC_ASSESSMENT","notes":"Child died from a massive intracranial bleed, probably due to Evan's syndrome and myeloablation; possible viral encephalitis possibly increased risk of intracranial hemorrhage.","organSystem":"Vascular disorders","stats":[{"groupId":"EG000","numAffected":1,"numAtRisk":35,"numEvents":1}],"term":"Massive intracranial bleed"}]}