Abstract:
:Deficiency of the CD40L, expressed on the surface of T lymphocytes, is caused by mutations in the glycoprotein CD40L (CD154) gene. Resulting defective humoral and cellular responses cause a clinical presentation that includes recurrent sinopulmonary bacterial infections, opportunistic infections, sclerosing cholangitis, neutropenia, and autoimmune manifestations. HSCT represents the only curative treatment modality. However, the therapeutic decision to use HSCT proves challenging in many cases, mainly due to the lack of a phenotype-genotype correlation. We retrospectively reviewed patients with CD40L deficiency who were transplanted in Antalya and Göztepe MedicalPark Pediatric HSCT units from 2014 to 2019 and followed by Akdeniz University School of Medicine Department of Pediatric Immunology. The records of eight male cases, including one set of twins, were evaluated retrospectively. As two transplants each were performed on the twins, a total of ten transplants were evaluated. Conditioning regimens were predominantly based on myeloablative protocols, except for the twins, who received a non-myeloablative regimen for their first transplantation. Median neutrophil and platelet engraftment days were 13 (range 10-19) and 14 (range 10-42) days, respectively. In seven of ten transplants, a CMV reactivation was developed without morbidity. None of the patients developed GVHD, except for one mild case of acute GVHD. All patients survived, and the median follow-up was 852 days. Our data show that HSCT for patients with CD40 ligand deficiency is a potentially effective treatment for long-term disease control.
journal_name
Pediatr Transplantjournal_title
Pediatric transplantationauthors
Uygun DFK,Uygun V,Karasu GT,Daloğlu H,Öztürkmen SI,Çelmeli F,Törün SH,Özen A,Barış S,Aydıner EK,Yalçın K,Kılıç SÇ,Hazar V,Bingöl A,Yeşilipek Adoi
10.1111/petr.13768subject
Has Abstractpub_date
2020-09-01 00:00:00pages
e13768issue
6eissn
1397-3142issn
1399-3046journal_volume
24pub_type
杂志文章abstract::Administration of hematopoietic growth factors, with or without chemotherapy, can augment progenitor cell numbers available for collection. The dose of granulocyte colony stimulating factor (G-CSF) used for mobilization of peripheral blood progenitor cells (PBPC) is controversial, and doses between 5 and 32 microg/kg/...
journal_title:Pediatric transplantation
pub_type: 临床试验,杂志文章,随机对照试验
doi:
更新日期:1998-05-01 00:00:00
abstract::Studies are increasingly recognizing health-related quality of life (HRQOL) as a key pediatric outcome in both clinical and research settings and an essential health outcome measure to assess the effectiveness of medical treatment. However, it has not yet been studied among the healthy siblings of kidney transplant re...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.13734
更新日期:2020-08-01 00:00:00
abstract::Although the majority of pediatric solid organ transplant centers in the United States employ psychosocial criteria to assess the suitability of potential transplant candidates, there are no standardized pretransplant psychosocial assessment measures. Assessment scales that have been developed were designed for adult ...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2007.00785.x
更新日期:2008-02-01 00:00:00
abstract::A five-yr-old boy developed chronic liver failure and ESKD because of CHF and juvenile NPHP. He underwent sequential liver and kidney transplantation with a compatible blood type from his father, at five yr, seven months and five yr, 11 months old, respectively. Because the patient was not in ESKD, we initially perfor...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2011.01611.x
更新日期:2012-11-01 00:00:00
abstract::To evaluate the role of BAL, CTB, and OLB in the management of pulmonary infiltrates in pediatric HSCT recipients, we conducted a retrospective review of clinical records of pediatric HSCT recipients. Data were analyzed using Chi-square for dichotomous and anova for continuous variables. Logistic regression was used t...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2007.00733.x
更新日期:2007-11-01 00:00:00
abstract::Facial transplantation has become a reality in adult medicine. Children are subject to congenital craniofacial differences, disease-related, traumatic, or thermochemical craniofacial changes and might be suitable for face transplantation. This manuscript addresses unique ethical issues in considering potential pediatr...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.13032
更新日期:2017-11-01 00:00:00
abstract::Nephrotic syndrome is characterized by proteinuria, hypoalbuminemia, and general edema. These symptoms may persist in children who reach ESRD, which is unfavorable for the patient's allograft outcome. In addition, this may hamper early diagnosis of a relapse after transplantation. Surgical bilateral nephrectomy is oft...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.13703
更新日期:2020-06-01 00:00:00
abstract::A proportion of patients with CF develop cirrhosis and portal hypertension. LT and combined LLT are rarely performed in patients with CF. To determine the outcome of LT and LLT in patients with CF. Patients with CF who had LT or LLT between 10/1987 and 5/2008 were identified from UNOS database. A total of 182 children...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2010.01460.x
更新日期:2011-05-01 00:00:00
abstract::Pharmacokinetic monitoring of CsA is unsatisfactory, because at comparable CsA blood concentrations, the frequency and severity of adverse effects vary considerably among patients. We have therefore recently developed a precise, reliable, and robust whole-blood pharmacodynamic assay that measures the suppression of Cs...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2010.01354.x
更新日期:2010-11-01 00:00:00
abstract::EBV is associated with various malignancies in patients with acquired or induced immune impairment. EBV-SMT is very uncommon in immunocompromised patients, and a kidney localization has been described only anecdotally. We report the case of a 17-yr-old kidney transplant recipient diagnosed as having an EBV-SMT inside ...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2009.01213.x
更新日期:2010-12-01 00:00:00
abstract::Sirolimus represents a major advancement in the field of solid organ transplantation and is being used as a rescue agent for acute and chronic rejection as well as for primary immunosuppression with good graft outcome. Although initial studies with sirolimus were in adults, now significant data have accumulated on the...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2005.00305.x
更新日期:2005-06-01 00:00:00
abstract::Retransplantation is often a necessity for children with end-stage renal disease (ESRD), as kidney graft survival is still not infinite. If a suitable live donor is present, the current policy is to use the live donor first, in order to obtain excellent long-term outcome and to prevent human leucocyte antigen (HLA) se...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1034/j.1399-3046.2001.00049.x
更新日期:2001-06-01 00:00:00
abstract::INFVA is an important cause of pulmonary infections in patients receiving BMT, and is associated with considerable morbidity and mortality for a readily preventable and treatable infection. Few studies have addressed the impact of the new neuraminidase inhibitors in the prognosis of influenza after BMT. The aim of thi...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2008.01114.x
更新日期:2010-03-01 00:00:00
abstract:UNLABELLED:Liver transplantation treats the hepatic affectation of UCDs; however, irreversible neurologic damage pretransplant is difficult to assess providing transplant teams with ethical dilemmas for liver transplantation. The purpose of our study was to determine whether pretransplant neuroimaging can predict devel...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.12520
更新日期:2015-08-01 00:00:00
abstract::Children who undergo heart transplantation are at risk for long-term neurodevelopmental sequelae secondary to heart disease and its treatment. Detailed neuropsychological profiles in clinical sample status post-pediatric heart transplantation are sparse in the literature, and there is little information regarding pred...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.13615
更新日期:2020-02-01 00:00:00
abstract::Methylmalonic acidemia with complete mutase deficiency (mut(0) type) is an inborn error of metabolism with high mortality and morbidity. LT has been suggested to be a solution to this disease, but elevation of urinary and blood MMA was still observed after LT. In this study, we measured dry blood spot MMA and its prec...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2009.01227.x
更新日期:2010-05-01 00:00:00
abstract::Dyslipidemia contributes to cardiovascular morbidity and mortality in pediatric transplant recipients. Data on prevalence and risk factors in pediatric cohorts are, however, scarce. We therefore determined the prevalence of dyslipidemia in 386 pediatric renal transplant recipients enrolled in the CERTAIN registry. Dat...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.12914
更新日期:2017-05-01 00:00:00
abstract::Acute liver failure (ALF) is a rare but often fatal disorder in childhood. Its aetiology includes infections, toxins, metabolic disorders, infiltrative diseases, autoimmune hepatitis, ischaemia, irradiation damage, but in a high proportion of cases it remains unknown. In contrast to adults, in children with ALF hepati...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2004.00292.x
更新日期:2004-12-01 00:00:00
abstract::The aim of the study was to demonstrate clinical course of the first reported cases of PLS in pediatric kidney transplantation and therapeutic outcome for such condition using a combination of high-dose corticosteroid and tacrolimus. We report a single case (a nine-year-old Thai boy) with end-stage kidney disease seco...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2006.00560.x
更新日期:2006-09-01 00:00:00
abstract::PTLD is a major complication after transplantation. Treatment options for PTLD are not standardized, usually sequential, starting with reduction in immunosuppression. Recently, we have used a dual combination of rituximab and reduced dose chemotherapy (R/C) directly after failed RI. We retrospectively identified 30 pe...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2010.01370.x
更新日期:2010-11-01 00:00:00
abstract::The purpose of this study was to assess the inter-rater reliability of the P-TRI, a 17-item instrument developed to identify risk factors associated with poor treatment adherence in pediatric solid organ transplant candidates. Because factors influencing treatment adherence may vary with age, the 89 subject samples we...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2010.01428.x
更新日期:2011-03-01 00:00:00
abstract::Organ transplants between genetically different individuals elicit powerful immune responses that invariably cause rejection in the absence of immune suppression. Among the immune responses elicited by organ allografts, B-cell responses causing antibody-mediated rejection are one of the most vexing. However, recent ad...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2007.00867.x
更新日期:2008-08-01 00:00:00
abstract::Although it is known that children undergoing heart transplantation are at increased risk for both AKI and CKD, renal function following CHLT remains understudied. All pediatric CHLT patients from 2006 to 2019 were included. The prevalence of AKI in the first 7 post-operative days, renal recovery at 30 post-operative ...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.13833
更新日期:2020-11-01 00:00:00
abstract::Tacrolimus (Tac)-related hypertrophic cardiomyopathy (HCM) has been reported to be an unusual but serious complication affecting pediatric patients after solid organ transplantation. Herein, we present a case of young liver transplant recipient with Tac-induced HCM, treated by discontinuation of Tac followed by conver...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2006.00633.x
更新日期:2007-05-01 00:00:00
abstract::From June 1985 to December 1998, 173 pediatric renal transplants were carried out in 170 patients at our center. From this pool, 73 patients (34 males and 39 females) with a follow-up of 48 months were examined. In all patients, ureteroneocystostomy was performed according to the Lich-Grégoire procedure. All patients ...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1034/j.1399-3046.1999.00017.x
更新日期:1999-08-01 00:00:00
abstract::The role of unrelated donor HSCT for children with de novo AML in CR1 is controversial. We performed this study to investigate the feasibility of unrelated donor HSCT who initially had intermediate- or high-risk cytogenetics. We retrospectively reviewed medical records of patients with AML who received unrelated HSCT ...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.13397
更新日期:2019-06-01 00:00:00
abstract::This study evaluated changes in growth parameters after pediatric heart transplantation and identified factors associated with the changes after pediatric heart transplantation (OHT). We retrospectively evaluated the somatic growth of 46 children <11 yr of age who underwent OHT for changes in weight, height, and BMI. ...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2007.00826.x
更新日期:2008-06-01 00:00:00
abstract:PURPOSE:The understanding of the HRQOL issues for parent donors of children who underwent LDLT is lacking. We evaluated the HRQOL of donor and non-donor parents, described their subjective experiences and identified factors associated with lower HRQOL post-donation. METHODS:This is a cross-sectional study of parent do...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.13337
更新日期:2019-03-01 00:00:00
abstract::Thrombosis is a major postoperative complication in pediatric liver transplantation. There is marked heterogeneity in prophylactic antithrombotic therapies used, without established guidelines. This review summarizes current worldwide incidence of thrombotic events and compares antithrombotic therapies in children pos...
journal_title:Pediatric transplantation
pub_type: 杂志文章,评审
doi:10.1111/petr.13420
更新日期:2019-06-01 00:00:00
abstract::Surgical techniques that have been used during liver transplantation (LT) together with patient's coagulation profile and institutional practices are reported to have an effect on transfusion requirements. The aim of this study is to evaluate the transfusion requirement in both cadaveric (CDLT, n = 22) and living dono...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2005.00284.x
更新日期:2005-06-01 00:00:00