ABSTRACT
A 6-year-old girl presented with recurrent skin rash at the initial stage, recent joint pain, and neutrophilia was found during a routine blood test. After a multidisciplinary case discussion, she was diagnosed with chronic neutrophil leukemia, and the symptoms were relieved after hydroxyurea and luxolitinib treatment. She received the allogeneic hematopoietic stem cell transplantation subsequently. At present, she is in stable condition and under follow-up. Chronic neutrophil leukemia is a rare disease, which rarely occurs in children. It is more difficult to diagnose in patients with skin rash as the first manifestation. The diagnosis and treatment of this case reflects the important role of multidisciplinary cooperation in the diagnosis and treatment of difficult and rare diseases.
ABSTRACT
Medulloblastoma is one of common brain tumor in childhood. Low risk patient has good response to convention therapy,such as operation,radiotherapy,but patients with high risk or recurrent patients get worse effect. Hematopoietic stem cell transplantation is an important therapy for refractory hematological disease and cancer,it has been used in high risk and recurrent medulloblastoma for decades. Now,the currant situation of hematopoietic stem cell transplantation in childhood medulloblastoma were summarized.
ABSTRACT
Objective To discuss thk curk kffkct and sidk kffkcts of donor anti-CD19 chimkric antigkn rkckp-tor T lemphocetks(CD19 CLA-T)for trkating rkcurrknt acutk B-ckll lkuckmia aftkr allogknkic hkmatopoiktic stkm-ckll transplantation(Lllo-HSCT),and to analezk thk influkncing factors for this thkrape. Methods Thk clinical data of 5 acutk B-ckll lkuckmia patiknts wkrk analezkd rktrospkctivkle who rklapskd aftkr Lllo-HSCT and rkckivkd donor CD19 CLA-T thkrape at Bkijing Childrkn＇s Hospital from Jule 2015 to Octobkr 2017. Diskask status bkfork infusion, conditioning rkgimkn,rkinfusion ckll dosk,and sidk-kffkct of CLA-T infusion,changks in thk rklatkd immunological indicators,and follow-up trkatmknt rksults wkrk invkstigatkd. ResuIts Onk patiknt had no kffkct,othkr patiknts got rk-mission or minimal rksidual diskask(MAD)nkgativk within 4 wkkcs aftkr CLA-T infusion,and thk middlk timk was 14 daes. Pkriphkral CLA-T pkac happknkd 2 wkkcs aftkr CLA-T infusion. Be thk last follow,2 patiknts dikd of lkuckmia, 3 patiknts wkrk alivk,and 1 cask of thkm livkd with tumor aftkr CD19 nkgativk rklapsk,othkrs livkd with diskask-frkk condition. Cetocink rklkask sendromk(CAS)was thk most common sidk kffkct,happkning in 1 to 2 wkkcs aftkr infusion, 1 patiknt had nkurologic toxicitiks,and 2 patiknts had suspicious graft -vkrsus -host diskask. ConcIusions Donor CD19 CLA-T thkrape has a good short-tkrm kffkct for rklapskd B -ckll lkuckmia patiknts aftkr Lllo -HSCT,but long-tkrm kffkct rkquirks furthkr obskrvation;CAS is thk most common sidk-kffkct. Off-targkt and ckll kxhaustion ark thk main rkasons for dkfkat.
ABSTRACT
Objective To analyze the clinical characteristics,treatment and prognosis of allogeneic hematopoietic stem cell transplantation (allo-HSCT)-associated thrombotic microangiopathy (TA-TMA) in children.Methods The clinical information,treatment and prognosis of 9 cases with TA-TMA hospitalized following alloHSCT from January 2008 to November 2017 in Hematology Oncology Center,Beijing Children's Hospital,Capital Medical University were retrospectively analyzed.Results Of all the 283 allo-HSCT recipients,9 patients (3.2％) were diagnosed as TA-TMA.Among them,there were 5 male and 4 female,with a median age of 94 months (39-129 months).The median time to of diagnosis was 63 days (6-342 days) after HSCT.Additionally,the median platelet counts,hemoglobin and lactate dehydrogenase(LDH) levels were 44 × 109/L [(7-75) × 109/L],76 g/L (40-105 g/L) and 594 U/L(445-1 386 U/L).Neurological symptoms were found in 5 of the patients,4 had kidney involvement,and 6 had gastrointestinal involvement.The major treatment of TA-TMA was plasma exchange,Rituximab and defibrotide instead of the use of calcineurin inhibitors.Finally,4 patients achieved response after treatment,5 children died of ineffective treatment.Conclusion TA-TMA is a fatal complication after allo-HSCT.It can lead to multiorgan and multi-systems dysfunction.If there are more than 2 systems involved in TA-TMA,it suggests poor prognosis.The combined therapy is better than monotherapy,and the selective individual treatment of TA-TMA is essential.