Sequential renal and bone marrow transplants in a child with Fanconi anemia.

Abstract:

:FA is an autosomal recessive disorder characterized by small stature and renal abnormalities. FA can lead to progressive bone marrow failure, myelodysplastic syndrome, or acute leukemia. Using a multidisciplinary team approach, we managed a 3-yr-old boy with FA who simultaneously developed renal and hematopoietic failure. Because renal function was insufficient to support the conditioning regimen for HCT, we performed a deceased donor renal transplant in December 2012 prior to HCT with the known risk of graft-versus-graft rejection of the donor kidney. Seven months later he underwent allogeneic HCT. He obtained myeloid engraftment on day +11 and peripheral blood chimerism demonstrated all donor by day +21. He developed asymptomatic CMV reactivation and despite antirejection medications, mild skin graft-versus-host disease. He has maintained excellent renal function and remains transfusion independent with full hematopoietic recovery. He has not experienced any renal rejection episodes nor developed donor-specific antibodies toward his renal donor. Peripheral blood chimerism remains completely HCT donor. He is clinically well, now greater than two and a half yr after renal transplant and two yr after HCT. The continuing close collaboration between the Pediatric Nephrology and Bone Marrow Transplant teams is a major factor in this successful outcome.

journal_name

Pediatr Transplant

authors

Vincent CL,Primack WA,Hipps J,Kasow KA

doi

10.1111/petr.12619

subject

Has Abstract

pub_date

2016-02-01 00:00:00

pages

146-50

issue

1

eissn

1397-3142

issn

1399-3046

journal_volume

20

pub_type

杂志文章
  • Lung transplantation and survival in children with cystic fibrosis: solid statistics--flawed interpretation.

    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

    authors: Sweet SC,Aurora P,Benden C,Wong JY,Goldfarb SB,Elidemir O,Woo MS,Mallory GB Jr,International Pediatric Lung Transplant Collaborative.

    更新日期:2008-03-01 00:00:00

  • The impact of thromboelastography on resuscitation in pediatric liver transplantation.

    abstract::Although TEG directs effective resuscitation in adult surgical patients, pediatric data are lacking. We performed a retrospective comparative review of the effect of TEG on blood product utilization and outcomes following pediatric liver transplantation in 38 patients between 2008 and 2014. Diagnoses, laboratory value...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章

    doi:10.1111/petr.13176

    authors: Sujka J,Gonzalez KW,Curiel KL,Daniel J,Fischer RT,Andrews WS,Wicklund BM,Hendrickson RJ

    更新日期:2018-06-01 00:00:00

  • Therapeutic drug monitoring of tacrolimus in pediatric liver transplant patients.

    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

    authors: MacFarlane GD,Venkataramanan R,McDiarmid SV,Pirsch JD,Scheller DG,Ersfeld DL,Fitzsimmons WE

    更新日期:2001-04-01 00:00:00

  • History of pediatric stem cell transplantation.

    abstract::During the past 50 yr, intensive studies into the use of hematopoietic cell transplantation (HCT) for therapy of cancer and non-malignant hematologic diseases have changed this treatment modality from one that was thought to be plagued by insurmountable complications to one that is now standard therapy for some diseas...

    journal_title:Pediatric transplantation

    pub_type: 历史文章,杂志文章

    doi:10.1111/j.1398-2265.2004.00203.x

    authors: Storb R

    更新日期:2004-06-01 00:00:00

  • Diet quality of children post-liver transplantation does not differ from healthy children.

    abstract::Little has been studied regarding the diets of children following LTX. The study aim was to assess and compare dietary intake and DQ of healthy children and children post-LTX. Children and adolescents (2-18 years) post-LTX (n=27) and healthy children (n=28) were studied. Anthropometric and demographic data and two 24-...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章

    doi:10.1111/petr.12944

    authors: Alzaben AS,MacDonald K,Robert C,Haqq A,Gilmour SM,Yap J,Mager DR

    更新日期:2017-09-01 00:00:00

  • Pediatric split liver transplantation after Fontan procedure in left isomerism combined with biliary atresia: a case report.

    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

    authors: Youn JK,Lee JM,Yi NJ,Choi YR,Suh SW,You T,Lee KW,Jung CW,Lee JW,Bae EJ,Ko JS,Kim WH,Park KW,Suh KS

    更新日期:2014-12-01 00:00:00

  • Pharmacokinetic analysis for model-supported therapeutic drug monitoring of busulfan in Japanese pediatric hematopoietic stem cell transplantation recipients.

    abstract::This prospective observational study analyzed the pharmacokinetics of busulfan in Japanese children and evaluated the predicting accuracy of previous pediatric PPK models of busulfan. This study enrolled five patients (aged 2-12 years, BW 14-48 kg) receiving a busulfan-based conditioning regimen for hematopoietic stem...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章

    doi:10.1111/petr.13696

    authors: Kishimoto K,Hasegawa D,Irie K,Okada A,Nakamura S,Tamura A,Yamamoto N,Kozaki A,Saito A,Ishida T,Fukushima S,Kosaka Y

    更新日期:2020-06-01 00:00:00

  • The SPLIT research agenda 2013.

    abstract::This review focuses on active clinical research in pediatric liver transplantation with special emphasis on areas that could benefit from studies utilizing the SPLIT infrastructure and data repository. Ideas were solicited by members of the SPLIT Research Committee and sections were drafted by members of the committee...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章,评审

    doi:10.1111/petr.12090

    authors: Alonso EM,Ng VL,Anand R,Anderson CD,Ekong UD,Fredericks EM,Furuya KN,Gupta NA,Lerret SM,Sundaram S,Tiao G,Studies of Pediatric Liver Transplantation (SPLIT) Research Group.

    更新日期:2013-08-01 00:00:00

  • Pediatric renal transplantation in the Nordic countries: a report of the Nordic Pediatric Renal Transplant Study Group.

    abstract::The Nordic Pediatric Renal Transplant Study Group was organized in 1994 to register and follow children under 16 years of age who receive renal allografts in the Nordic countries. It comprises all transplant centers in Scandinavia, four in Sweden, three in Denmark and one each in Norway and Finland. The following repo...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章,多中心研究

    doi:

    authors: Tydén G,Berg U

    更新日期:1998-08-01 00:00:00

  • The effects of gender on health-related quality of life in pediatric live-donor kidney transplantation: a single-center experience in a developing country.

    abstract::To evaluate the effects of gender on HRQOL and overall health status in our pediatric kidney transplants. We performed a cross-sectional study in 77 children who received living renal allo-transplants in our center between 1981 and 2003. The patients were given a questionnaire at a post-transplant visit. After complet...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章

    doi:10.1111/j.1399-3046.2009.01174.x

    authors: El-Husseini A,Hassan R,Sobh M,Ghoneim M

    更新日期:2010-03-01 00:00:00

  • VPS 45-associated primary infantile myelofibrosis--successful treatment with hematopoietic stem cell transplantation.

    abstract::PMF of infancy is a recently described autosomal recessive disorder presenting with severe bone marrow failure, accelerated neutrophil apoptosis, and significant platelet dysfunction, caused by a mutation in the VPS45 gene. In this study, we update our group of patients with PMF, highlighting different aspects of this...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章

    doi:10.1111/petr.12169

    authors: Stepensky P,Simanovsky N,Averbuch D,Gross M,Yanir A,Mevorach D,Elpeleg O,Weintraub M

    更新日期:2013-12-01 00:00:00

  • Controlling the blood glucose of type 1 diabetes mice by co-culturing MIN-6 β cells on 3D scaffold.

    abstract::T1D is an autoimmune disease, which may be caused by lack of insulin-secreting β cells due to damage of autoimmune system. Living with T1D is a challenge for the child and the family; cell transplantation is a treatment option for diabetes in children. To establish a microenvironment suitable for cell growth and proli...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章

    doi:10.1111/petr.12443

    authors: Yuan X,Huang Y,Guo Y,Wang L,Guo Q,Xu T,Wu D,Zhou P,Zhu S,Wang Y,Fan X,Zhu M,Lu Y,Wang Z

    更新日期:2015-06-01 00:00:00

  • Impact of growth hormone treatment on a Belgian population of short children with renal allografts.

    abstract::We retrospectively analyzed the effects of recombinant human growth hormone (rhGH) in a Belgian population of 36 short children with renal allografts. Seven children were dropped from the growth study: 1 had skeletal dysplasia and in 6 cases rhGH was given for less than 1 yr (1 died, 1 developed genu valgum, 2 were no...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章

    doi:

    authors: Janssen F,Van Damme-Lombaerts R,Van Dyck M,Hall M,Schurmans T,Herman J,Hooghe L,Van Damme B

    更新日期:1997-11-01 00:00:00

  • Denying renal transplantation to an adolescent medical cannabis user: An ethical case study.

    abstract::Medical cannabis is now legal in over half of the United States. As more patients adopt this unconventional therapy, it is inevitable that potential transplant recipients will disclose their cannabis use during transplant evaluation. Transplant teams are tasked with the decision to utilize a pressure resource, often w...

    journal_title:Pediatric transplantation

    pub_type:

    doi:10.1111/petr.13467

    authors: Ryan JE,Noeder M,Burke C,Stubblefield SC,Sulieman S,Miller EG

    更新日期:2019-08-01 00:00:00

  • Multiple rare-earth magnet bead ingestion in a pediatric liver-small bowel-pancreas transplant recipient: A case report and lessons learned.

    abstract::Ingestion of rare-earth magnet beads in children has been a public health concern. The potential risk of swallowing multiple magnets is related to magnet attraction to each other, resulting in serious gastrointestinal complications, such as entero-enteric fistula formation, peritonitis, bowel ischemia or necrosis, bow...

    journal_title:Pediatric transplantation

    pub_type:

    doi:10.1111/petr.13915

    authors: Miri A,Nakayuenyongsuk W,Vargas L,Langnas A,Vo HD

    更新日期:2020-11-20 00:00:00

  • Pediatric post-transplant hepatic kaposi sarcoma due to donor-derived human herpesvirus 8.

    abstract::In areas of the world where human herpesvirus 8 (HHV-8) is endemic, Kaposi sarcoma (KS) is a common SOT-associated cancer. In the United States, where the virus is not prevalent, PTKS is rare, and there is little literature on pediatric PTKS. We present a North American female who underwent deceased donor, left latera...

    journal_title:Pediatric transplantation

    pub_type:

    doi:10.1111/petr.13384

    authors: Ocwieja KE,Vargas SO,Elisofon SA,Shulman DS,Lee CK,Fawaz R,Collins N,Vakili K,Sharma TS

    更新日期:2019-05-01 00:00:00

  • Approaches to acute liver failure in children.

    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

    authors: Dhawan A,Cheeseman P,Mieli-Vergani G

    更新日期:2004-12-01 00:00:00

  • Grading acute graft-versus-host disease: time to reconsider.

    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

    authors: Goyal RK,Goyal M,Sankaranarayan K

    更新日期:2015-05-01 00:00:00

  • Combined liver-kidney transplantation for primary hyperoxaluria type I in children: Single Center Experience.

    abstract::Primary hyperoxalurias are rare inborn errors of metabolism with deficiency of hepatic enzymes that lead to excessive urinary oxalate excretion and overproduction of oxalate which is deposited in various organs. Hyperoxaluria results in serious morbid-ity, end stage kidney disease (ESKD), and mortality if left untreat...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章

    doi:10.1111/petr.13313

    authors: Kotb MA,Hamza AF,Abd El Kader H,El Monayeri M,Mosallam DS,Ali N,Basanti CWS,Bazaraa H,Abdelrahman H,Nabhan MM,Abd El Baky H,El Sorogy STM,Kamel IEM,Ismail H,Ramadan Y,Abd El Rahman SM,Soliman NA

    更新日期:2019-02-01 00:00:00

  • Invasive diagnostic procedures for pulmonary infiltrates in pediatric hematopoietic stem cell transplant recipients.

    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

    authors: Armenian SH,Hoffman JA,Butturini AM,Kapoor N,Mascarenhas L

    更新日期:2007-11-01 00:00:00

  • Reno- and splenoportal anastomosis for a retransplant patient with situs inversus.

    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

    authors: Uchida H,Fukuda A,Sasaki K,Shigeta T,Hirata Y,Kanazawa H,Mali VP,Miyazaki O,Nosaka S,Sakamoto S,Mureo K

    更新日期:2016-06-01 00:00:00

  • Non-adherence in pediatric liver transplant recipients--an assessment of risk factors and natural history.

    abstract::Despite the fact that non-adherence to medical therapy is one of the major causes of late morbidity and mortality in pediatric liver transplant recipients, little is known of the risk factors involved in this behavior. Three cases of fatal non-adherence are reported. Factors associated with non-adherence were investig...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章

    doi:10.1034/j.1399-3046.2000.00110.x

    authors: Lurie S,Shemesh E,Sheiner PA,Emre S,Tindle HL,Melchionna L,Shneider BL

    更新日期:2000-08-01 00:00:00

  • The role of continuous renal replacement therapy in the management of acute kidney injury associated with sinusoidal obstruction syndrome following hematopoietic cell transplantation.

    abstract::Maintaining fluid balance, pre- and post-MA-HCT is essential and usually requires frequent administration of diuretics. Hepatic sinusoidal obstructive syndrome is potentially life-threatening, especially when associated with AKI and MOF. This study describes six patients who developed AKI-associated SOS and diuretic-r...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章

    doi:10.1111/petr.13139

    authors: Raina R,Abusin GA,Vijayaraghavan P,Auletta JJ,Cabral L,Hashem H,Vogt BA,Cooke KR,Abu-Arja RF

    更新日期:2018-03-01 00:00:00

  • Successful ABO incompatible renal transplantation following rituximab and DFPP after failed immunoadsorption.

    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

    authors: Thalgahagoda S,Webb NJ,Roberts D,Birch A,Milford DV,Tavakoli A,Shenoy M

    更新日期:2014-05-01 00:00:00

  • Lipoprotein profile changes in children after renal transplantation in the modern immunosuppression era.

    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

    authors: Sgambat K,He J,McCarter RJ,Moudgil A

    更新日期:2008-11-01 00:00:00

  • Successful recovery of aplastic anemia following orthotopic liver transplantation for non-A-E acute liver failure.

    abstract::This report describes a teenager who developed aplastic anemia (AA) because of non-A-E acute liver failure (ALF) requiring orthotopic liver transplantation (OLT). His AA did not recover spontaneously and he required treatment with ATG 9 months post-OLT. Bone marrow recovery occurred 4 months after immunotherapy and co...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章

    doi:10.1111/j.1399-3046.2005.00321.x

    authors: Rajwal SR,Davison SM,Stringer MD,Kinsey SE,McClean P

    更新日期:2005-08-01 00:00:00

  • Resistance to growth hormone in children with chronic liver disease.

    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:

    authors: Bucuvalas JC,Horn JA,Chernausek SD

    更新日期:1997-08-01 00:00:00

  • Cognitive and emotional outcome after pediatric liver transplantation.

    abstract::The aim of the study was to evaluate the cognitive and emotional development after pediatric liver transplantation. A total of 21 patients, aged 4-16.9 yr (median 9.6 yr) were tested 1-9 yr (median 4.2 yr) after the transplantation. The pretransplant diagnoses included biliary atresia (eight patients), various metabol...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章

    doi:10.1034/j.1399-3046.2003.00081.x

    authors: Adebäck P,Nemeth A,Fischler B

    更新日期:2003-10-01 00:00:00

  • A national conference to determine research priorities in pediatric solid organ transplantation.

    abstract::The need for evidence-based practice guidelines requires high quality, carefully controlled clinical research trials. This multidisciplinary conference attempted to: identify urgent clinical and research issues, identify obstacles to performing clinical trials, develop concepts for organ-specific and all-organ researc...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章

    doi:10.1111/j.1399-3046.2007.00811.x

    authors: Bartosh SM,Ryckman FC,Shaddy R,Michaels MG,Platt JL,Sweet SC

    更新日期:2008-03-01 00:00:00

  • Reversal of tacrolimus-related hypertrophic cardiomyopathy after conversion to rapamycin in a pediatric liver transplant recipient.

    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

    authors: Turska-Kmieć A,Jankowska I,Pawłowska J,Kaliciński P,Kawalec W,Tomyn M,Markiewicz M,Teisseyre J,Czubkowski P,Rekawek J,Socha J

    更新日期:2007-05-01 00:00:00