Abstract:
:Alternate donor HSCT for thalassemia major from a matched unrelated donor or haploidentical family donor is a feasible therapeutic option in children with no matched family donor. Aggressive pretransplant immunosuppression, reduced toxicity conditioning, and PTCY result in excellent thalassemia-free survival. We describe here our experience in this cohort. We performed a retrospective analysis of the data on children who underwent a haploidentical HSCT for thalassemia major with PTCY at our center from August 2017 to August 2019. All children received pretransplant immune suppression for 6 weeks with fludarabine and dexamethasone, hypertransfusion and chelation with intravenous desferrioxamine. Conditioning included thiotepa, fludarabine, rabbit ATG, and cyclophosphamide, and GvHD prophylaxis included PTCY with tacrolimus. Twenty children were included and nineteen children engrafted. Acute hypertension occurred in five children, bacterial infection in eight children and viral respiratory infection in three children. Three children suffered from graft rejection. Reactivation of viruses namely CMV, adenovirus, and BK virus was seen in 60% of children. Grades 1-2 acute GvHD of the skin in four children (20%) and limited chronic GvHD of the skin in four children (20%). Immune cytopenia was documented in three children (15%). Haploidentical HSCT offers a therapeutic option for children with thalassemia major with no suitably matched family or unrelated donors. Our reduced toxicity regimen with PTCY offers a DFS of 75% and OS of 95% with low transplant-related mortality of 5%.
journal_name
Pediatr Transplantjournal_title
Pediatric transplantationauthors
Vellaichamy Swaminathan V,Ravichandran N,Ramanan KM,Meena SK,Varla H,Ramakrishnan B,Jayakumar I,Uppuluri R,Raj Rdoi
10.1111/petr.13893subject
Has Abstractpub_date
2020-10-27 00:00:00pages
e13893eissn
1397-3142issn
1399-3046pub_type
杂志文章abstract::Children receiving HCT loose protective immunity to vaccines received pre-HCT. Therefore, revaccination post-HCT is of major importance. In Denmark, a vaccination schedule with fewer doses post-HCT has been used, including two doses for diphtheria, tetanus, polio, measles, mumps, and rubella, and one dose only for Hae...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.13599
更新日期:2020-02-01 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::FH is a hereditary inherited disorder of cholesterol metabolism. Homozygous form of the disease associates severe form of atherosclerotic disease. Clinicians have been tried to inhibit the progression of the homozygous FH with medical and surgical treatment. We here present three siblings with homozygous FH who were s...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2010.01469.x
更新日期:2011-05-01 00:00:00
abstract::Activation of the RAS has a crucial role in the progression of ischemia/reperfusion-associated CAD. The regulation of RAS differs in the two genders. However, the extent of gender differences and locations of renin production have not been revealed yet. We investigated in vivo the local renin production in the two gen...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.12712
更新日期:2016-08-01 00:00:00
abstract::Total body irradiation and high-dose chemotherapy, applied as a preparatory regimen for bone marrow transplantation (BMT) in children with acute lymphoblastic leukemia (ALL), are particularly hazardous to the gonads and, in addition, can impair hypothalamo pituitary-gonadal control. Longitudinal data on pubertal devel...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1034/j.1399-3046.1999.00006.x
更新日期:1999-02-01 00:00:00
abstract:BACKGROUND:Heart transplant waitlist mortality remains high in infants <1 year of age and among those with CHD. Currently, the median accepted donor-to-recipient weight percentage is approximately 130% of the recipient's weight. We hypothesized that patients with CHD may accept a larger organ using novel 3D-derived ima...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.13639
更新日期:2020-02-01 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::Acute graft-versus-host disease (GVHD) is a common and serious complication of allogeneic blood and marrow transplantation. Acute GVHD is commonly graded according to modified Glucksberg criteria. There is considerable within-grade heterogeneity with different patterns of skin, liver, or gut involvement. In this comme...
journal_title:Pediatric transplantation
pub_type: 杂志文章,评审
doi:10.1111/petr.12433
更新日期:2015-05-01 00:00:00
abstract::While 24-h ambulatory blood pressure monitoring (ABPM) is an established tool for monitoring antihypertensive therapy in adults, data in children are scarce. We retrospectively analysed whether office blood pressure (BP) is reliable for the diagnosis of BP control in 26 treated hypertensive paediatric renal transplant...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2006.00595.x
更新日期:2007-02-01 00:00:00
abstract::The majority of transplant centers around the world face an ethical debate whether to retransplant a young non-adherent patient. Non-adherence to lifelong immunosuppressants presents a significant risk for graft loss, yet rates remain consistently high. Despite a number of these patients presenting for retransplantati...
journal_title:Pediatric transplantation
pub_type: 杂志文章,评审
doi:10.1111/j.1399-3046.2011.01633.x
更新日期:2012-02-01 00:00:00
abstract::Depression is common in pediatric chronic kidney disease (CKD) patients. Depression is associated with inferior long-term outcomes. There is a paucity of studies that evaluate depression and possible associated factors in children and adolescents requiring renal replacement therapy (RRT). Cross-sectional study using C...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.13591
更新日期:2019-12-01 00:00:00
abstract::In the pediatric population, little is known on de novo DSA development, its impact on graft function, and association with suboptimal IS. We assessed the prevalence of de novo DSA in the Vienna cohort of 40 renal transplanted children and adolescents and prospectively followed its association with clinical parameters...
journal_title:Pediatric transplantation
pub_type: 临床试验,杂志文章
doi:10.1111/petr.12707
更新日期:2016-06-01 00:00:00
abstract:: FSGS has a high recurrence rate after renal transplantation. To examine the effects of the use of preemptive and post-transplant PP on recurrence and graft outcome, we conducted a retrospective study on 34 pediatric patients (mean age 13±5 yr) with biopsy-proven pretransplant FSGS and who underwent a renal transplan...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2011.01478.x
更新日期:2011-08-01 00:00:00
abstract::PV thrombosis following pediatric LT is a serious complication that may lead to graft loss. LDLT poses limitations with regard to the availability of vein grafts for complex PV reconstructions. We herein report a unique reconstruction of the PV inflow in a one-yr-old boy with situs inversus undergoing re-LDLT. The inf...
journal_title:Pediatric transplantation
pub_type:
doi:10.1111/petr.12703
更新日期:2016-06-01 00:00:00
abstract:BACKGROUND:Scientific advancements are occurring in cardiac xenotransplantation (XTx). However, there have been religious and social concerns surrounding this allotransplantation alternative. The purpose of this study was to explore the acceptance of XTx among stakeholders of the congenital heart disease (CHD) communit...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.13851
更新日期:2020-10-06 00:00:00
abstract::De novo PTAID may develop in pediatric solid organ transplant recipients, have a diverse spectrum, and are occasionally treatment resistant. Previous reports showed resolution of immune cytopenias in solid organ transplant recipients following replacement of the calcineurin inhibitor tacrolimus with the mTOR inhibitor...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.13737
更新日期:2020-09-01 00:00:00
abstract::There are little data on prevalence of dyslipidemia in pediatric kidney TX recipients in the modern IS era. LP profiles of 38 TX recipients receiving triple IS with MMF, prednisone, and tacrolimus were compared with those of 11 children on HD using mixed model multiple linear regression analysis of repeated measures a...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2008.00905.x
更新日期:2008-11-01 00:00:00
abstract::aHUS is caused by the over-activation and dysregulation of the alternative complement pathway. Data regarding outcomes of pediatric aHUS patients after kidney transplantation are still very scarce. Accordingly, the aim of this study was to describe the clinical findings and outcomes of pediatric aHUS patients after re...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.13914
更新日期:2020-11-20 00:00:00
abstract::Pneumatosis intestinalis is an uncommon finding beyond the neonatal period, but it has been reported in immunocompromized pediatric patients. The association of pneumatosis intestinalis in children following renal transplantation has to the best of our knowledge been only reported once in children. We describe a 4-yea...
journal_title:Pediatric transplantation
pub_type: 杂志文章,评审
doi:10.1034/j.1399-3046.2003.00061.x
更新日期:2003-06-01 00:00:00
abstract::Children who develop end-stage renal disease (ESRD) as a result of obstructive uropathies require evaluation and treatment of associated bladder dysfunction to ensure a good outcome following renal transplantation. Bladder dynamics can often be optimized medically, although surgical intervention is occasionally necess...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1034/j.1399-3046.2002.01078.x
更新日期:2002-06-01 00:00:00
abstract::Chronic rejection is the leading cause of graft loss following pediatric kidney transplantation. Our group and others have demonstrated an association between the development of Abs to self-antigens and chronic rejection following adult lung and heart transplantation. The goal of this study was to determine whether Ab...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.12531
更新日期:2015-08-01 00:00:00
abstract::We report on survival, rejection, lymphoma and renal function following cardiac transplant using a steroid-free maintenance immunosuppressive regimen. We have performed 73 cardiac transplants in 71 children under 16 yr of age in the last 12 yr. There were eight perioperative and four late deaths giving actuarial survi...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1034/j.1399-3046.2003.00014.x
更新日期:2003-02-01 00:00:00
abstract::The clinical utility of tacrolimus monitoring in adults has been well documented. The present study compared tacrolimus monitoring in a pediatric population of 34 liver transplant patients in four US centers with an adult population of 111 patients in six US centers. Subjects (adult and pediatric) were evaluated, at d...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1046/j.1397-3142.2000.00000.x
更新日期:2001-04-01 00:00:00
abstract::HSCT is associated with a high risk of late morbidity. The aim of this study was to evaluate the frequency, time frame, risk factors, and possible etiology of pulmonary dysfunction following allogeneic HSCT in childhood. We evaluated the pulmonary function of 51 HSCT patients (>6 yr), by including FVC and FEV1 values ...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.12313
更新日期:2014-09-01 00:00:00
abstract::Vascular complications post-renal transplantation are not very common and can be associated with increased risk of graft loss. We report an unusual case of a young, female teenager with asymmetrical leg swelling. After one month of transplantation, the patient developed a rise in serum creatinine with right leg swelli...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2008.00941.x
更新日期:2008-09-01 00:00:00
abstract::Knowledge concerning the involvement of the cardiovascular system in children awaiting liver transplant is limited. Therefore, no guidelines have been established on evaluating this group of patients for cardiac disease. This review examines the diverse cardiovascular manifestations of liver disease in children. We al...
journal_title:Pediatric transplantation
pub_type: 杂志文章,评审
doi:10.1111/j.1399-3046.2012.01667.x
更新日期:2012-06-01 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::Malnutrition adversely affects mortality and morbidity before and after liver transplantation. Outcome might be improved if liver transplant recipients were in a better nutritional state at the time of transplantation. In this review, we will examine the potential use of GH and IGF-I to improve nutritional status in p...
journal_title:Pediatric transplantation
pub_type: 杂志文章,评审
doi:
更新日期:1997-08-01 00:00:00
abstract::There are few studies on invasive pneumococcal disease in pediatric transplant recipients. Given this fact plus the advent of pneumococcal conjugate vaccines, we conducted a retrospective study at a major pediatric transplant center. The objectives were to determine the incidence and outcomes of invasive pneumococcal ...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2005.00275.x
更新日期:2005-04-01 00:00:00
abstract::Omenn's syndrome is a rare inherited variant of SCID. It is inevitably fatal, unless treated by bone marrow or stem cell transplantation. However, treatment-related complications and graft rejection are major obstacles to the success of transplantation. In this report, we describe an eight-month-old baby with Omenn's ...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2010.01413.x
更新日期:2012-03-01 00:00:00