Clinical Research Condition Of Beta-thalassemia Major

Objective: In order to understand the present situation of theBeta-thalassemia major (TM) with Clinical treatment, to find the causeof the failed to standardize treatment,and put forward interventionmeasures, thus improving the quality of survival and life of patients, byanalysising the clinical manifestations and the treatment process of TM.Methods: Retrospective analysis30cases of TM hospitalized duringJan2005to Aug2012in Women’s and Children’s central Hospital ofChengdu and36cases of TM hospitalized during Jan2002to Jan2012inChildren’s Hospital of Chongqing Medical University. The clinicalmanisfestations, age of onset, laboratory examination,diagnostic method,and process of treatment.Results:1.66cases all were diagnosed according to《The diagnosis andtreatment guidelines of Beta-thalassemia major》.12cases were diagnosedon the basis of beginning Hb、HbF and the family history and54caseswere diagnosed on the basis of the genetic diagnosis. 2.There were39cases of male,27cases of female, and the sex ratiowas1.44:1.3.The onset age of patients ranged from less than1month to1years’ old.25.8%patients were1month,80.3%were less than6months.4.The diagnosis of age of patients ranged from2months to3years’old.80.3%patients were less than1years’ old,19.7%were more thanyears’ old.5.66cases were from different distribution:29cases come fromChengdu,36cases come from Chongqing, and1case come from Guizhou.38cases come from countryside,28cases come from the city. The ratioof countryside and city was1.36:1.6.There were13cases of height to achieve the median,23casesof weight to achieve the median,and11cases of both height andweight to achieve the median.7.There were1cases with diabetes mellitus,1case heart failure,3case with autoimmunehemolyticanaemia,4cases with hypersplenism,25cases liver function damage and22cases with Anaemic sex heart disease.8.12cases which were diagnosed on the basis of beginning Hb，HbF and the family history, there are12cases Hb <60g/L; there are4cases HbF between70%and80%,4cases HbF between80%and90%,and4cases HbF>90%.9.54cases who confirmed based on the genetic diagnosis included 16cases homozygous, accounting for29.6%;38cases doubleheterozygote, accounting for70.3%.10.There were4cases had undergone a bone marrow transplant.Atpresent,3cases have no need blood transfusion, and1case also need bloodtransfusion.11.3cases had undergone splenectomy for hypersplenism. Because theage of undergoing splenectomy in3cases were more than5years old,sountil now,there were no serious infection after splenectomy in3cases.12.There were4cases who had canonical blood transfusion and irontherepy,10cases who had canonical blood transfusion,but not hadcanonical iron therepy,3cases who had canonical iron therepy,but not hadcanonical blood transfusion,46cases who had not canonical iron therepyand canonical blood transfusion,and3cases who had no need bloodtransfusion.13.12cases of66cases had received deferiprone to iron therapy.20cases of66cases had received deferoxamine to iron therapy.11cases of46cases had received deferiprone combined deferoxamine to iron therapy.3cases of66cases had received deferasirox to iron therapy.14.4cases with canonical blood transfusion and iron therepy hadserum ferritin levels between1000-1500μg/L.10cases withcanonical blood transfusion but no canonical iron therepy had serumferritin levels between2000-5000μg/L.4cases with no canonical blood transfusion but canonical iron therepy had serum ferritin levelsbetween1000-1500μg/L.49cases without canonical blood transfusionand canonical iron therepy had serum ferritin levels more than8000μg/L.Conclusion:1. This research shows that the onset age of Beta-thalassemia majorwas early.The onset age of patients ranged from1month to1years’ old.25.8%patients were1month,80.3%were less than6months.2. Our data show that the incidence ratio between urban and ruralchildren was1.36:1, a higher incidence in rural children.3. The incidence of developmental disorders was high in patients ofBeta-thalassemia major, accounting for83.3%(55/66).4.Beta-thalassemia major can merge diabetes and autoimmunehemolytic anemia.5.Splenectomy was a effective treatment for Beta-thalassemia majorwith hypersplenis. After Splenectomy it can extend the transfusioninterval, and reduce the amount of blood transfusion.6.This research shows that patients who had specification of bloodtransfusion and to iron treated had long-term survival. This study alsoshowed the timing of iron treateddepends on serum ferritin level.