Abstract:
:CEP is a rare inborn error of porphyrin-heme synthesis. Clinical manifestations can range from mild to severe and include erythrodontia, reddish-colored urine, and hemolytic anemia that can be mild or severe and may result in splenomegaly. Completely avoiding exposure to the sun is crucial. Attempts to reduce erythropoiesis and to lower circulating porphyrin levels by means of erythrocyte transfusions have been successful in reducing the expression of the disease. However, the complications of a chronic transfusion regimen are potentially severe. Successful bone marrow transplantation has been reported in CEP. We report a case of successful bone marrow transplantation and prolonged follow-up in an adolescent CEP patient.
journal_name
Pediatr Transplantjournal_title
Pediatric transplantationauthors
Faraci M,Morreale G,Boeri E,Lanino E,Dallorso S,Dini G,Scuderi F,Cohen A,Cappelli Bdoi
10.1111/j.1399-3046.2007.00842.xsubject
Has Abstractpub_date
2008-02-01 00:00:00pages
117-20issue
1eissn
1397-3142issn
1399-3046pii
PTR842journal_volume
12pub_type
杂志文章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::ADV is frequently seen in our pediatric SOT population. It presents in a variety of clinical presentation and can cause severe disease. In this population, there are very few studies to determine the safety of CDV as a potential therapeutic agent. We present the findings of our retrospective study evaluating the effic...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.13231
更新日期:2018-06-03 00:00:00
abstract::Clinical grading of GI involvement during acute GVHD remains a challenging issue, especially in children. Plasma citrulline, a non-protein amino acid selectively produced and released by enterocytes, is a suitable surrogate endpoint for small intestinal epithelial cell mass, irrespective of the underlying cause of cel...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.12128
更新日期:2013-11-01 00:00:00
abstract::Vascular complications remain the most common cause of early renal allograft loss in patients with end-stage renal failure. Underlying thrombophilic disorders increase the risk of early graft thrombosis. A male adolescent with high-risk thrombophilia because of combined heterozygous factor V Leiden (G1691A) and prothr...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2005.00398.x
更新日期:2006-02-01 00:00:00
abstract::Organ transplant is life-saving and any given organ may be valuable to a multitude of potential recipients. An allocation system must be used to reconcile the difference between supply and demand, and this system must take into account the impact that accepting international patients may have on the local system. The ...
journal_title:Pediatric transplantation
pub_type: 杂志文章,评审
doi:10.1111/petr.12769
更新日期:2016-09-01 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::We reviewed 112 pediatric renal transplant recipients to document the rate of medication non-adherence (NA) and to examine the relationships between NA, comorbid psychiatric illness, and the outcome variables of acute and chronic rejection and graft loss. A total of 32.5% of subjects had clinically significant NA with...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1046/j.1397-3142.2003.00117.x
更新日期:2003-12-01 00:00:00
abstract::For infants with SCID, the ideal conditioning regimen before allogeneic HCT would omit cytotoxic chemotherapy to minimize short- and long-term complications. We performed a prospective pilot trial with G-CSF plus plerixafor given to the host to mobilize HSC from their niches. We enrolled six patients who received CD34...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.12309
更新日期:2014-09-01 00:00:00
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::We assessed our long-term experience with regards to the safety and efficacy of MMF in our pediatric renal transplant population and compared it retrospectively to our previous non-MMF immunosuppressive regimen. Forty-seven pediatric renal transplants received MMF as part of their immunosuppressive protocol in the per...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2009.01179.x
更新日期:2011-05-01 00:00:00
abstract::Vitamin C deficiency in developed countries is typically observed in patients with unique clinical conditions such as cystic fibrosis or anorexia nervosa, or in patients on long-term tube feeds. We report here a clinical observation in six pediatric and adolescent patients (median age 17.5 yr, range 9.8-23.5 yr) with ...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.12285
更新日期:2014-08-01 00:00:00
abstract::In their provocative paper, "Lung transplantation and survival in children with cystic fibrosis," Liou and colleagues state that "Prolongation of life by means of lung transplantation should not be expected in children with cystic fibrosis. A prospective, randomized trial is needed to clarify whether and when patients...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2008.00924.x
更新日期:2008-03-01 00:00:00
abstract::The prospective cross-sectional study investigated the 6MWT performance in pediatric group of liver transplant recipients (6-17 yr, median post-transplantation time of 22 months) and compared to the normal values obtained in healthy children as well as evaluated the reproducibility of the 6MWT. We analyzed the relatio...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.12318
更新日期:2014-09-01 00:00:00
abstract::Management of late humoral rejection remains challenging, and DSA may persist. A case report illustrates how individual DSA titers using solid-phase-based assays may help to assess for accommodation. A male cystinosis patient received a cadaveric renal transplant at the age of 12 yr with a daclizumab, tacrolimus, MMF,...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.12242
更新日期:2014-06-01 00:00:00
abstract::LI is a subset of the heterotaxy syndrome and a rare birth defect that involves the heart and other organs. It can be combined with extracardiac abnormalities, especially BA. CHD can be associated with LI in up to 15% of cases, although it is rare in BA. Pediatric LT for a child with ESLD due to BA combined with LI an...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.12364
更新日期:2014-12-01 00:00:00
abstract::Recurrent and de novo disease is an increasing problem and is known to negatively impact transplant graft survival. Immunosuppressive medications have not had an impact on the prevalence of recurrent and de novo disease. Renal Allograft Disease Registry (RADR) was established to study the prevalence, impact and risk f...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2004.00180.x
更新日期:2004-08-01 00:00:00
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 descr...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.13893
更新日期:2020-10-27 00:00:00
abstract::LT is an effective therapeutic option for a variety of IEM. This approach can significantly improve the quality of life of patients who suffer from severe disease manifestations and/or life-threatening metabolic decompensations despite medical/dietary management. Due to the significant risks for systemic complications...
journal_title:Pediatric transplantation
pub_type: 杂志文章,评审
doi:10.1111/petr.12741
更新日期:2016-09-01 00:00:00
abstract::BWS is one of the most well-known somatic overgrowth syndromes, which is characterized by macroglossia, organomegaly, abdominal wall defects, and predisposition to embryonal tumors, such as Wilms' tumor, hepatoblastoma, and adrenocortical carcinoma. We report a case of BWS in a girl with unresectable hepatoblastoma, w...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/j.1399-3046.2009.01198.x
更新日期:2010-11-01 00:00:00
abstract::Despite advances in therapy for acute leukemia, relapse continues to be the major cause of treatment failure. Hematopoietic stem cell transplant can rescue some patients after relapse, but the ability to escalate the intensity of preparative regimens is limited by toxicity to normal organs. Radiolabeled monoclonal ant...
journal_title:Pediatric transplantation
pub_type: 杂志文章,评审
doi:10.1034/j.1399-3046.7.s3.14.x
更新日期:2003-01-01 00:00:00
abstract::This report describes a successful renal Tx in a patient with chronic renal failure, caused by dysplastic kidneys, who received a cadaveric kidney with post-infectious glomerulonephritis. Sequential renal biopsies were performed at 12 h before Tx, and at 1 h and on days 8 and 58 post-Tx. Post-operative hematuria disap...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1034/j.1399-3046.2000.00076.x
更新日期:2000-02-01 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::Effective antibody removal using PE, DFPP and IA has led to increased access to live donor organs through ABOi RT for patients with chronic kidney disease. However, there have been no head-to-head comparator studies between these modalities, and the choice of technique is usually influenced by cost and institutional p...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.12227
更新日期:2014-05-01 00:00:00
abstract::Autoimmune and allergic diseases cause morbidity and diminished quality of life in pediatric organ transplant recipients. We hypothesize that younger age at transplantation and immunosuppression regimen play a role in the development of immune-mediated disease following heart transplant. A single institution retrospec...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.12205
更新日期:2014-03-01 00:00:00
abstract::Children (defined as under 18 yr of age) account for approximately 12.5% of all liver transplants in the United States. Even though the annual number of liver transplantation procedures remains relatively constant, the population of long-term survivors of liver transplantation has grown. Presently, the population of l...
journal_title:Pediatric transplantation
pub_type: 杂志文章,评审
doi:10.1034/j.1399-3046.2002.1r058.x
更新日期:2002-02-01 00:00:00
abstract::Bacillus cereus is a spore-forming, gram-positive bacterium that causes food poisoning presenting with either emesis or diarrhea. Diarrhea is caused by proteinaceous enterotoxin complexes, mainly hemolysin BL, non-hemolytic enterotoxin (NHE), and cytotoxin K. In contrast, emesis is caused by the ingestion of the depsi...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.12378
更新日期:2015-02-01 00:00:00
abstract::RTx of adult-size kidneys presents a size mismatch in small pediatric recipients, and there are potential surgical complications. This study reveals the outcomes of intra- and extraperitoneal RTx in low-weight (less than 15 kg) pediatric recipients. We studied 51 pediatric patients weighing less than 15 kg who receive...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.13401
更新日期:2019-06-01 00:00:00
abstract::The high discard rate of pediatric donor hearts presents a major challenge for children awaiting heart transplantation. Recent literature identifies several factors that contribute to the disparities in pediatric donor heart usage, including regulatory oversight, the absence of guidelines on pediatric donor heart acce...
journal_title:Pediatric transplantation
pub_type: 杂志文章,评审
doi:10.1111/petr.13655
更新日期:2020-05-01 00:00:00
abstract::Few studies have identified the psychosocial characteristics of those children and their families associated with future non-adherence to immunosuppressive medications following a heart or lung transplant. UNOS data and medical records information were used to test the association between patient and family psychosoci...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.12899
更新日期:2017-06-01 00:00:00
abstract::LDLT is a well-established treatment for most terminal liver diseases in children. Survival rates have improved, yet few studies have considered HRQoL or sleep problems in LDLT recipients. In this cross-sectional study, we enrolled 51 children who had undergone LDLT in Renji Hospital. PedsQL(™) 4.0 Generic Core Scales...
journal_title:Pediatric transplantation
pub_type: 杂志文章
doi:10.1111/petr.12532
更新日期:2015-08-01 00:00:00