ABSTRACT
Objective:To investigate the prognosis of childhood adrenoleukodystrophy (ALD) with cognitive disorder after haploidentical allogenic hematopoietic stem cell transplantation (haplo-HSCT), and to identify risk factors affecting the prognosis.Methods:It was a single-center retrospective study involving 31 ALD children receiving haplo-HSCT in Peking University People′s Hospital from January 2014 to October 2022.Survival analysis was performed by Kaplan-Meier method. Cox regression analysis was performed to identify risk factors for the prognosis of childhood ALD following haplo-HSCT. Results:Among the 31 children with ALD, 1 case died of cardiogenic shock during the transplantation, and the remaining had a successful haplo-HSCT.Ten children with ALD had cognitive disorder before haplo-HSCT, including 3 cases with the minimal LOES score ≥10 points and 8 cases with the Neurologic Function Score (NFS)>0 point before haplo-HSCT.Six children had major functional disability (MFD) and 2 cases died due to progression of ALD after haplo-HSCT.Twenty children did not have cognitive disorder before haplo-HSCT, of whom 3 cases had the LOES score≥10 points and 6 cases had NFS>0 before haplo-HSCT.Four children had MFD and 2 cases died due to progression of ALD after haplo-HSCT.For ALD patients without cognitive disorder after haplo-HSCT, the 3-year and 5-year survival rate were 100.0% and 72.9%, respectively, and the 5-year MFD-free survival was 61.6%.For ALD patients with cognitive disorder after haplo-HSCT, the 3-year survival rate was 83.3%.Compared with ALD patients with the LOES score<10 points before haplo-HSCT, those with the LOES score≥10 points had 9.243 times the risk of developing MFD after haplo-HSCT ( P=0.024, 95% CI: 1.332-64.127). Compared with ALD patients without cognitive disorder before haplo-HSCT, ALD patients with cognitive disorder had 9.749 times the risk of developing MFD after haplo-HSCT ( P=0.023, 95% CI: 1.358-66.148). Conclusions:Cognitive disorder and LOES score≥10 points before haplo-HSCT are risk factors for developing MFD in children with ALD following haplo-HSCT.
ABSTRACT
【Objective】 To investigate the clinical outcomes of allogeneic hematopoietic stem cell transplantation in elderly patients with acute leukemia and the role of comprehensive nursing. 【Methods】 The data of 52 elderly patients with acute leukemia during the treatment of allogeneic hematopoietic stem cell transplantation were collected. According to the characteristics of elderly patients, 52 patients were given comprehensive nursing measures such as psychological, protective isolation, dietary management and specialized nursing. Stem cell engraftment, transplant complications and survival rates were observed in patients with comprehensive nursing support. 【Results】 All patients received comprehensive care. Of the 52 patients, 49 (94.2%) achieved neutrophil engraftment, with a median engraftment time of 12 days (9~19 days), and 45 patients (86.5%) achieved platelet engraftment with a median engraftment time of 13 days (9~35 days). The cumulative incidence of Ⅱ-Ⅳ°acute graft-versus-host disease (GVHD) was 26.9%, and the cumulative incidence of chronic GVHD was 28.5%. Cytomegalovirus (CMV) infection occurred in 15 cases, with a cumulative incidence rate of 37.5%, and Epstein-Barr virus (EBV) infection occurred in 5 cases, with a cumulative incidence rate of 9.9%. Bloodstream bacterial infection occurred in 8 patients with a cumulative incidence of 9.6%. With a median follow-up of 226 days (71~2 365 days), 39 patients survived and 13 died. The 1-year overall survival (OS) was 71.4%, and the OS and disease free survival (DFS) of patients with negative minimal residual disease (MRD) were both 100%. OS and DFS in MRD positive group were 59.2% and 48.1%, respectively. 【Conclusion】 Elderly patients with acute leukemia who receive allogeneic hematopoietic stem cell transplantation can also achieve long-term survival. Taking targeted preventive and nursing measures can ensure the success rate of transplantation.
ABSTRACT
Introducción: La aplasia medular adquirida grave es una enfermedad hematológica infrecuente caracterizada por una disminución o ausencia de precursores hematopoyéticos en la médula ósea, lo cual se expresa con distintos grados de citopenias. Varios factores, infecciosos o no, pueden incidir en su origen. Su manejo es complejo y puede incluir tratamiento inmunosupresor y trasplante de progenitores hematopoyéticos alogénico. Objetivo: Demostrar la utilidad de la realización del trasplante de progenitores hematopoyéticos alogénico haploidéntico en pacientes con aplasia medular grave. Caso clínico: Paciente masculino de 21 años de edad, con antecedentes de salud, que en octubre del 2018 debutó con íctero, pancitopenia, lesiones purpúrico hemorrágicas en piel y mucosas, en el curso de una hepatitis aguda seronegativa. La biopsia de médula ósea mostró aplasia medular severa. Se inició tratamiento inmunosupresor con globulina antitimocίtica, ciclosporina A y metilprednisolona. Al cabo de los 6 meses mantenía trombocitopenia severa con necesidades transfusionales y en octubre de 2019 se decide realizar trasplante de progenitores hematopoyéticos alogénico con donante haploidéntico y empleando como tratamiento acondicionante globulina antitimocίtica, fludarabina, ciclofosfamida y bajas dosis de irradiación corporal total. En evaluación clίnica de julio de 2020 (dίa + 280 del trasplante) el paciente estaba asintomático y con parámetros hematológicos normales. Conclusiones: Se demostró que el trasplante de progenitores hematopoyéticos alogénico haploidéntico es un proceder realizable y útil en pacientes con aplasia medular grave, lo cual corrobora el beneficio clínico que puede aportar su ejecución en pacientes con esta enfermedad(AU)
Introduction: Acquired severe marrow aplasia is a rare hematological disease characterized by decrease or absence of hematopoietic precursors in bone marrow, which is expressed with different degrees of cytopenias. Several factors, infectious or not, can influence its origin. Its management is complex and may include immunosuppressive treatment and allogeneic hematopoietic stem-cell transplantation. Objective: To demonstrate the usefulness of performing haploidentical allogeneic hematopoietic stem-cell transplantation in patients with severe medullary aplasia. Clinical case: A 21-year-old male patient, with medical history, who first presented, in October 2018, with icterus, pancytopenia, as well as purpuric hemorrhagic lesions on the skin and mucosa, in the course of acute seronegative hepatitis. The bone marrow biopsy showed severe marrow aplasia. Immunosuppressive treatment was started with antithymocytic globulin, cyclosporine A, and methylprednisolone. After six months, he maintained severe thrombocytopenia under transfusion requirements and, in October 2019, the decision was to perform allogeneic hematopoietic stem-cell transplantation with a haploidentical donor and using antithymocyte globulin, fludarabine, cyclophosphamide, and low doses of total body irradiation as conditioning treatment. In the clinical assessment carried out in July 2020 (day +280 after transplantation), the patient was asymptomatic and with normal hematological parameters. Conclusions: Transplantation of haploidentic allogeneic hematopoietic progenitors was shown to be a feasible and useful procedure in patients with severe marrow aplasia, which corroborates the clinical benefit that its execution can bring in patients with this disease(AU)
Subject(s)
Humans , Male , Young Adult , Tissue Donors/ethics , Methylprednisolone/therapeutic use , Whole-Body Irradiation/methods , Microscopy, Electron, Scanning Transmission/methods , Hematologic Diseases , Hematopoietic Stem Cell Transplantation/methods , Cuba , Transplantation, Haploidentical/methods , Anemia, Aplastic/therapy , Antilymphocyte SerumABSTRACT
BACKGROUND: Forkhead box P3 (FOXP3) is an important marker of regulatory T cells. FOXP3 polymorphisms are associated with autoimmune diseases, cancers, and allograft outcomes. We examined whether single nucleotide polymorphisms (SNPs) at the FOXP3 locus are associated with clinical outcomes after allogenic hematopoietic stem cell transplantation (HSCT). METHODS: Five FOXP3 SNPs (rs5902434, rs3761549, rs3761548, rs2232365, and rs2280883) were analyzed by PCR-sequencing of 172 DNA samples from allogenic HSCT patients. We examined the relationship between each SNP and the occurrence of graft-versus-host disease (GVHD), post-HSCT infection, relapse, and patient survival. RESULTS: Patients with acute GVHD (grades II-IV) showed higher frequencies of the rs3761549 T/T genotype, rs5902434 ATT/ATT genotype, and rs2232365 G/G genotype than did patients without acute GVHD (P=0.017, odds ratio [OR]=5.3; P=0.031, OR=2.4; and P=0.023, OR=2.6, respectively). Multivariate analysis showed that the TT genotype of rs3761549 was an independent risk factor for occurrence of acute GVHD (P=0.032, hazard ratio=5.6). In contrast, the genotype frequencies of rs3761549 T/T, rs5902434 ATT/ATT, and rs2232365 G/G were lower in patients with post-HSCT infection than in patients without infection (P=0.026, P=0.046, and P=0.031, respectively). CONCLUSIONS: rs3761549, rs5902434, and rs2232365 are associated with an increased risk of acute GVHD and decreased risk of post-HSCT infection.
Subject(s)
Humans , Allografts , Autoimmune Diseases , DNA , Genotype , Graft vs Host Disease , Hematopoietic Stem Cell Transplantation , Hematopoietic Stem Cells , Multivariate Analysis , Odds Ratio , Polymorphism, Single Nucleotide , Recurrence , Risk Factors , T-Lymphocytes, RegulatoryABSTRACT
Kidney is rarely an involved organ of graft-versus-host disease (GVHD). Here, we report on a case of membranous nephropathy and interstitial nephritis after allogenic hematopoietic stem cell transplantation (HSCT) in a 44-year-old female patient with acute lymphoblastic leukemia. The patient received GVHD prophylaxis with low dose steroid, cyclosporin, and short course methotrexate. Cyclosporine was tapered out 17 months after allogeneic HSCT. Thereafter, the patient developed kidney impairment and nephrotic range proteinuria. Kidney biopsy revealed membranous nephropathy concurrent with interstitial nephritis.