Comparison of melphalan- And busulfan-based myeloablative conditioning in children undergoing allogeneic transplantation for acute myeloid leukemia or myelodysplasia.


BACKGROUND:The optimal conditioning regimen for alloHCT in children with myeloid malignancies remains undefined. PROCEDURE:We performed a retrospective review of children undergoing alloHCT for AML and MDS over a 10-year period (2008-2018) at our institution, comparing the outcomes of recipients of either a myeloablative busulfan- or reduced toxicity mel/thio-based conditioning regimen. RESULTS:A total of 49 patients underwent alloHCT for AML/MDS (mel/thio, N = 21; busulfan, N = 28). Mel/thio recipients were selected due to pretransplant comorbidities. Recipients of mel/thio were more likely to have t-AML, and less likely to have MRD <0.1% at the time of alloHCT (57.1% vs 82.1%). Graft failure was more common in busulfan recipients; engraftment kinetics were similar between groups. Sinusoidal obstructive syndrome was diagnosed in 21% of busulfan and no mel/thio recipients (P = .03). One patient in each group died from TRM. Relapse incidence was comparable (mel/thio-29% vs busulfan-32%); however, relapse occurred significantly later in recipients of mel/thio conditioning (median d + 396 vs d + 137; P = .01). As a result, there was a trend toward improved OS at 1 and 3 years in mel/thio recipients (95% vs 74%, P = .06; and 75% vs 50%, P = .11; respectively). CONCLUSION:In our single institution, when compared to myeloablative busulfan-based conditioning, use of a mel/thio-based reduced toxicity regimen resulted in comparable outcomes, despite higher risk patient and disease characteristics. Mel/thio recipients had both more comorbidities and higher risk disease profile, which did not translate into higher rates of either TRM or relapse.


Pediatr Transplant


Oshrine B,Adams L,Nguyen ATH,Amankwah E,Shyr D,Hale G,Petrovic A




Has Abstract


2020-06-01 00:00:00












  • Non-invasive biological quantification of acute gastrointestinal graft-versus-host disease in children by plasma citrulline.

    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


    authors: Merlin E,Minet-Quinard R,Pereira B,Rochette E,Auvrignon A,Oudot C,Sapin V,Deméocq F,Kanold J

    更新日期:2013-11-01 00:00:00

  • New insights into the functions of B cells.

    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


    authors: Balin SJ,Platt JL,Cascalho M

    更新日期:2008-08-01 00:00:00

  • Successful umbilical cord blood hematopoietic stem cell transplantation in pediatric patients with MDS/AML associated with underlying GATA2 mutations: two case reports and review of literature.

    abstract::Germline GATA2 mutations have been associated with a vast array of clinical manifestations, as well as hematological deficiencies and a propensity to AML or MDS. We present two cases of pediatric AML/MDS with underlying GATA2 mutations who underwent a successful umbilical cord hematopoietic stem cell transplantation u...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章,评审


    authors: Mallhi K,Dix DB,Niederhoffer KY,Armstrong L,Rozmus J

    更新日期:2016-11-01 00:00:00

  • Option of pre-emptive nephrectomy and renal transplantation for Bartter's syndrome.

    abstract::Bartter's syndrome (BS) is an incurable genetic disease, with variable response to supportive therapy relating to fluid and electrolyte management. Poor control or therapy non-compliance may result in frequent life threatening episodes of dehydration, acidosis and hypokalemia, with resultant adverse effects on patient...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Chaudhuri A,Salvatierra O Jr,Alexander SR,Sarwal MM

    更新日期:2006-03-01 00:00:00

  • Latin American Registry of Pediatric Renal Transplantation 2004-2008.

    abstract:UNLABELLED:The Latin American Pediatric Nephrology Association (ALANEPE) reports the first regional kidney transplant registry in Latin America. A mean range of 75%, 30% to 100% renal transplant recipients under 21 yr old were included. Out of 20 countries invited to participate, 14 performed renal transplant, 11 had u...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Latin American Pediatric Nephrology Association.,Latin American Pediatric Renal Transplant Cooperative Study.

    更新日期:2010-09-01 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


    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

  • Donor-specific HLA antibodies in paediatric cardiac transplant recipients are associated with poor graft survival.

    abstract::There is increasing evidence that DSA are associated with poor graft survival, although there are little data in children. We aimed to describe the incidence of DSA in this group and to determine correlation with graft survival. HLA antibodies were analysed in 59 paediatric cardiac transplant recipients. Mean age 10.4...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Irving C,Carter V,Parry G,Hasan A,Kirk R

    更新日期:2011-03-01 00:00:00

  • Recurrence of non-alcoholic steatohepatitis after liver transplantation in a 13-yr-old boy.

    abstract::Nonalcoholic steatohepatitis (NASH) is the most severe form of non-alcoholic fatty liver disease (NAFLD). The aim of our study was to highlight NASH as a rare but possible problem in children. We present a case of 13-yr-boy with a well-established diagnosis of liver cirrhosis secondary to NASH, who underwent orthotopi...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Jankowska I,Socha P,Pawlowska J,Teisseyre M,Gliwicz D,Czubkowski P,Kalicinski P,Cielecka-Kuszyk J,Socha J

    更新日期:2007-11-01 00:00:00

  • Successful allogeneic stem cell transplant for leukocyte adhesion deficiency using an adjusted busulfan-containing regimen.

    abstract::LAD is a rare and fatal congenital disorder in which there is a defect of the leukocyte adhesion molecule (CD18) on neutrophils. Severe LAD results in frequent and potentially fatal infections. Although allogeneic HSCT is the only curative treatment for severe LAD, strategies for HSCT remain to be established since a ...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Yoshimoto A,Hashii Y,Kashiwagi H,Koizumi M,Tokimasa S,Fujisaki H,Ohta H,Hanai N,Ozono K,Hara J

    更新日期:2007-06-01 00:00:00

  • Plasma cell-rich acute cellular rejection of a transplanted kidney associated with antibody to the red cell Kidd antigen.

    abstract::Plasma cell-rich acute cellular rejection of a transplanted kidney is described in association with the identification of serum antibody to the red cell Kidd antigen, Jk-b, which is also found in the kidney. This antibody was formed without a history of a recent blood transfusion or exposure to intravenous immunoglobu...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Hamilton MS,Singh V,Warady BA

    更新日期:2006-12-01 00:00:00

  • Cardiac magnetic resonance imaging in children with chronic kidney disease and renal transplantation.

    abstract::CV disease is the major cause of death in patients with CKD. Recently, CMR imaging emerges as a complementary method providing advantages in cardiac assessment; however, data on CMR in pediatric CKD are scarce. We performed CMR in 15 children: two with CKD, six on peritoneal dialysis, seven on hemodialysis, and in 18 ...

    journal_title:Pediatric transplantation

    pub_type: 临床试验,杂志文章


    authors: Schaefer B,Rusai K,Toth A,Pasti K,Ujszaszi A,Kreko M,Horvath E,Sallay P,Reusz GS,Merkely B,Tulassay T,Szabo AJ

    更新日期:2012-06-01 00:00:00

  • Simultaneous surgical and interventional radiological approach to treat complicated biliary strictures after pediatric liver transplantation.

    abstract::Post-transplantation biliary strictures occur in 5-15% of the pediatric liver transplant patients and are conventionally managed by interventional radiological techniques. Failure of this treatment leads to reoperation and sometimes to retransplantation. Herein, we describe a surgical approach and interventional radio...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Salvalaggio PR,Bambini DA,Donaldson J,Saker M,Whitington PF,Alonso EM,Superina RA

    更新日期:2004-10-01 00:00:00

  • Liver transplantation for neonatal hemochromatosis: analysis of the UNOS database.

    abstract::NH is the most common identifiable cause of ALF in the neonate. LT is the definitive treatment for neonates with NH who have failed medical therapy. Our aim was to determine the outcomes of LT in infants with NH. Patients (less than one yr of age) with NH who were listed for LT and patients who underwent LT between 19...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Sheflin-Findling S,Annunziato RA,Chu J,Arvelakis A,Mahon D,Arnon R

    更新日期:2015-03-01 00:00:00

  • Long-term follow-up in adult living donors for combined liver/bowel transplant in pediatric recipients: a single center experience.

    abstract::Pediatric candidates for combined liver/bowel transplant (LBTx) experience a very high mortality on the cadaver waiting list. Our transplant center has successfully used adult living donors to treat pediatric candidates for LBTx. We report the long-term follow-up of this unique cohort of organ donors. The charts of si...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Ghafari JL,Bhati C,John E,Tzvetanov IG,Testa G,Jeon H,Oberholzer J,Benedetti E

    更新日期:2011-06-01 00:00:00

  • ABO-incompatible deceased donor pediatric liver transplantation: Novel titer-based management protocol and outcomes.

    abstract::ABO-ILT have re-emerged as an alternate option for select patients awaiting transplant. However, treatment protocols for children undergoing deceased donor ABO-ILT are not standardized. We implemented a novel IS protocol for children undergoing deceased donor ABO-ILT based on pretransplant IH titers. Children with hig...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Mysore KR,Himes RW,Rana A,Teruya J,Desai MS,Srivaths PR,Zaruca K,Calvert A,Guffey D,Minard CG,Morita E,Hensch L,Losos M,Kostousov V,Hui SR,Orange JS,Goss JA,Nicholas SK

    更新日期:2018-11-01 00:00:00

  • Monitoring and modulation of Epstein-Barr virus loads in pediatric transplant patients.

    abstract::A major risk faced by bone-marrow and solid organ transplant patients is the development of post-transplant lymphoproliferative disease or post-transplant lymphoma (PTLD). In pediatric transplantation, PTLD onset is often associated with a rapid rise in Epstein-Barr virus (EBV) load in peripheral blood mononuclear cel...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Orentas RJ,Schauer DW Jr,Ellis FW,Walczak J,Casper JT,Margolis DA

    更新日期:2003-08-01 00:00:00

  • Severe cryptosporidiosis in a seven-year-old renal transplant recipient: case report and review of the literature.

    abstract::Cryptosporidium is an intracellular protozoa that can cause gastroenteritis in humans. In immunocompromised hosts, infection can be severe, leading to life-threatening persistent diarrhea. There is limited experience in treating this infection in solid organ transplants. Although newer drugs active against Cryptospori...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章,评审


    authors: Hong DK,Wong CJ,Gutierrez K

    更新日期:2007-02-01 00:00:00

  • Liver retransplantation in an infant requiring cavoportal hemi transposition.

    abstract::Historically inability to achieve portal inflow to the liver allograft operatively was felt to be a contraindication to orthotopic liver transplantation (OLTx). Cavoportal hemi transposition has been utilized more recently in adult OLTx recipients but rarely in pediatric recipients. Here we report the case of a 10-mon...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Verran D,Crawford M,Stormon M,Shun A

    更新日期:2004-08-01 00:00:00

  • Medication adherence in pediatric renal transplant patients: The role of family functioning and parent health locus of control.

    abstract::Children and adolescents with renal disease experience daily social, emotional, and medical challenges. Renal transplantation can help to improve quality of life but requires a lifelong regimen of immunosuppressant medication to maintain health. Adherence to a daily complex regimen can be difficult, particularly for a...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Kraenbring MM,Zelikovsky N,Meyers KEC

    更新日期:2019-03-01 00:00:00

  • An unusual case of anemia in a cardiac transplant recipient.

    abstract::Anemia is a well-described comorbidity in patients with heart failure and has been associated with decreased survival rates after heart transplant. The causes of anemia are broad, and identification of the underlying etiology is critical for management. Herein, we report an unusual case of severe anemia complicating c...

    journal_title:Pediatric transplantation



    authors: Lehto E,Raj A,Sparks J

    更新日期:2018-06-01 00:00:00

  • Potential influence of tacrolimus and steroid avoidance on early graft function in pediatric renal transplantation.

    abstract::With the increasing adoption of steroid-sparing immunosuppression protocols in renal transplantation, it is important to evaluate any adverse effects of steroid avoidance on graft function. Early graft function, measured by CrCl was retrospectively studied in 158 consecutive pediatric renal transplant recipients from ...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Li L,Weintraub L,Concepcion W,Martin JP,Miller K,Salvatierra O,Sarwal MM

    更新日期:2008-09-01 00:00:00

  • Health-related quality of life, clinical outcomes, and subjective experiences of parent donors of pediatric liver transplant recipients.

    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


    authors: Yuen WY,Tan A,Karthik SV,Quak SH,Kow AW,Aw MM

    更新日期:2019-03-01 00:00:00

  • Antithrombotic management and thrombosis rates in children post-liver transplantation: A case series and literature review.

    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


    authors: Flynn E,Huang JY,Hardikar W,Herd L,Hodgson A,Monagle P

    更新日期:2019-06-01 00:00:00

  • Impact of pretransplant splenectomy on patients with beta-thalassemia major undergoing a matched-related allogeneic stem cell transplantation.

    abstract::Impact of pretransplant splenectomy in patients with beta-thalassemia major undergoing an allogeneic SCT has never been addressed. Twenty-seven class III patients (29 transplants) had a pretransplant splenectomy. The outcome of these 29 transplants was compared with 76 transplants in class III who did not have a splen...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Mathews V,George B,Lakshmi KM,Viswabandya A,John JM,Sitaram U,Daniel D,Chandy M,Srivastava A

    更新日期:2009-03-01 00:00:00

  • Health-related quality of life among siblings of kidney transplant recipients.

    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


    authors: Velasco J,Ferraris JR,Eymann A,Ghezzi LFR,Coccia PA,Ferraris V

    更新日期:2020-08-01 00:00:00

  • Health-related quality of life and sleep among Chinese children after living donor liver transplantation.

    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


    authors: He K,Shen C,Chen X,Han L,Xi Z,Zhou T,Zhang J,Xia Q

    更新日期:2015-08-01 00:00:00

  • Risk for post-transplant lymphoproliferative disorder after polyclonal antibody induction in kidney transplantation.

    abstract::The adjusted relative risk (aRR) for development of post-transplant lymphoproliferative disorder (PTLD) is higher in kidney transplant recipients receiving monoclonal antibody induction therapy, but the aRR between the different available polyclonal agents has not been investigated in detail. We analyzed data from the...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Dharnidharka VR,Stevens G

    更新日期:2005-10-01 00:00:00

  • Liver transplantation for pediatric inherited metabolic disorders: Considerations for indications, complications, and perioperative management.

    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


    authors: Oishi K,Arnon R,Wasserstein MP,Diaz GA

    更新日期:2016-09-01 00:00:00

  • Pediatric discard risk index for predicting pediatric liver allograft discard.

    abstract:BACKGROUND:Of the 600 pediatric candidates added to the liver waiting list annually, 100 will remain waiting while over 100 liver allografts are discarded, often for subjective reasons. METHODS:We created a risk index to predict discard to better optimize donor supply. We used the UNOS database to retrospectively anal...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Malik T,Joshi M,Godfrey E,Galvan T,O'Mahony CA,Cotton R,Goss J,Rana A

    更新日期:2021-01-06 00:00:00

  • Primary HHV 6 infection after liver transplantation with acute graft rejection and multi-organ failure: successful treatment with a 2.5-fold dose of cidofovir and reduction of immunosuppression.

    abstract::HHV type 6 has been reported with enhanced pathogenicity in immunocompromised patients. Herein, we report about a two-yr-old girl who experienced primary HHV 6 infection after liver transplantation. She clinically presented with graft rejection and necrotic hepatitis as well as high fever, pneumonitis with respiratory...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Dohna-Schwake C,Fiedler M,Gierenz N,Gerner P,Ballauf A,Breddemann A,Läer S,Baba HA,Hoyer PF

    更新日期:2011-09-01 00:00:00