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












  • Outcomes of Berlin Heart EXCOR® pediatric ventricular assist device support in patients with restrictive and hypertrophic cardiomyopathy.

    abstract::The outcomes of pediatric ventricular assist device support in patients with diastolic heart failure have not been well described. This study reviews the North American experience with Berlin Heart EXCOR® ventricular assist device implants in children with such physiology. The Berlin Heart clinical database was review...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Su JA,Menteer J

    更新日期:2017-12-01 00:00:00

  • Using individual DSA titers to assess for accommodation after late humoral rejection.

    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


    authors: Filler G,Grimmer J,Ball E,Sharma AP,Huang SH

    更新日期:2014-06-01 00:00:00

  • Living donor liver transplantation for hepatoblastoma with Beckwith-Wiedemann syndrome.

    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


    authors: Sasaki K,Kasahara M,Fukuda A,Shigeta T,Tanaka H,Nakagawa S,Mitsui K,Harada R,Nakagawa A

    更新日期:2010-11-01 00:00:00

  • Anellovirus loads are associated with outcomes in pediatric lung transplantation.

    abstract::Anelloviruses are DNA viruses ubiquitously present in human blood. Due to their elevated levels in immunosuppressed patients, anellovirus levels have been proposed as a marker of immune status. We hypothesized that low anellovirus levels, reflecting relative immunocompetence, would be associated with adverse outcomes ...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Blatter JA,Sweet SC,Conrad C,Danziger-Isakov LA,Faro A,Goldfarb SB,Hayes D Jr,Melicoff E,Schecter M,Storch G,Visner GA,Williams NM,Wang D

    更新日期:2018-02-01 00:00:00

  • Focal segmental glomerulosclerosis associated with acute cytomegalovirus infection in a renal transplant.

    abstract::Focal segmental glomerulosclerosis (FSGS) occurring in association with cytomegalovirus (CMV) infection in a renal transplant patient with no previous history of FSGS has rarely been reported. We present a case of a 16-year-old renal transplant recipient who developed acute hepatitis, leukopenia, nephrotic syndrome, a...

    journal_title:Pediatric transplantation



    authors: Wynd E,Stewart A,Burke J

    更新日期:2019-09-01 00:00:00

  • Neuroradiologic findings in children with renal transplantation under 5 years of age.

    abstract::Chronic renal failure (CRF) is known to have adverse effects on the neurodevelopmental outcome of affected children. Some of these effects can be ameliorated by transplantation (TX). The cause and nature of the underlying brain injury is not known. We performed a brain magnetic resonance imaging (MRI) study on a group...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Valanne L,Qvist E,Jalanko H,Holmberg C,Pihko H

    更新日期:2004-02-01 00:00:00

  • Pediatric cardiac waitlist mortality-Still too high.

    abstract::Cardiac transplantation for children with end-stage cardiac disease with no other medical or surgical options is now standard. The number of children in need of cardiac transplant continues to exceed the number of donors considered "acceptable." Therefore, there is an urgent need to understand which recipients are in ...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章,评审


    authors: Denfield SW,Azeka E,Das B,Garcia-Guereta L,Irving C,Kemna M,Reinhardt Z,Thul J,Dipchand AI,Kirk R,Davies RR,Miera O

    更新日期:2020-05-01 00:00:00

  • Expanding the donor pool for congenital heart disease transplant candidates by implementing 3D imaging-derived total cardiac volumes.

    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


    authors: Shugh SB,Szugye NA,Zafar F,Riggs KW,Villa C,Lorts A,Morales DLS,Moore RA

    更新日期:2020-02-01 00:00:00

  • Chronic Mycoplasma pneumoniae infection in a child after renal transplantation.

    abstract::Mycoplasma pneumoniae has rarely been reported in renal transplant recipients. We present the case of a 10-yr-old boy with a six-month history of chronic cough, recurrent pyrexia, and weight loss three yr after RTx. The patient's post-transplant course was complicated by recurrence of NS that resolved with plasmaphere...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Schwerk N,Hartmann C,Baumann U,Pape L,Ehrich JH,Hansen G

    更新日期:2010-05-01 00:00:00

  • Preparing for a kidney transplant: Medical nephrectomy in children with nephrotic syndrome.

    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


    authors: Vos E,Koster-Kamphuis L,van de Kar NCAJ,Bootsma-Robroeks CMHHT,Cornelissen EAM,Schreuder MF

    更新日期:2020-06-01 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


    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

  • 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

  • Use of a virtual community as a psychosocial support system in pediatric transplantation.

    abstract::Despite significant interest by pediatric transplant patients in meeting others who have undergone transplantation, geographic distances combined with their daily routines make this difficult. This mixed-method study describes the use of Zora, a Web-based virtual community designed to create a support system for these...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Bers MU,Beals LM,Chau C,Satoh K,Blume ED,DeMaso DR,Gonzalez-Heydrich J

    更新日期:2010-03-01 00:00:00

  • Genetic variation and hematopoietic stem cell transplantation: expansion of the paradigm.

    abstract::Genetic variation has been the mainstay of hematopoietic stem cell transplantation since the first transplants were attempted. A significant expansion of genetic knowledge is under way, as the draft sequence of the human genome is annotated. Hematopoietic stem cell transplantation is a field that will greatly benefit ...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Savage SA,Chanock S

    更新日期:2003-01-01 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

  • Adolescent classroom education on knowledge and attitudes about deceased organ donation: a systematic review.

    abstract::In many countries, adolescents can choose to register a deceased organ donation wish when they apply for a driver's license. They often receive education about deceased organ donation in order to make an informed choice. The objective of this review was to describe the effectiveness of school-based educational program...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章,评审


    authors: Li AH,Rosenblum AM,Nevis IF,Garg AX

    更新日期:2013-03-01 00:00:00

  • Donor-specific HLA antibodies and graft function in kidney-transplanted children - the Vienna cohort.

    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


    authors: Rusai K,Dworak J,Potemkina A,Fischer G,Csaicsich D,Arbeiter K,Aufricht C,Müller-Sacherer T

    更新日期:2016-06-01 00:00:00

  • High depression rates among pediatric renal replacement therapy patients: A cross-sectional study.

    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


    authors: Rodriguez Cuellar CI,García de la Puente S,Hernández Moraria J,Bojórquez Ochoa A,Filler G,Zaltzman Grishevich S

    更新日期:2019-12-01 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

  • Evaluation of cardiac manifestations in pediatric liver transplant candidates.

    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


    authors: Madan N,Arnon R,Arnon R

    更新日期:2012-06-01 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

  • 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

  • A systematic review of immunosuppressant adherence interventions in transplant recipients: Decoding the streetlight effect.

    abstract::Non-adherence to immunosuppressant medications is an important risk factor for graft dysfunction. To evaluate the effectiveness of adherence-enhancing interventions, we reviewed adherence intervention studies in solid organ transplant recipients (all ages). Using the following databases: PsycINFO, PubMed, Scopus, and ...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章,评审


    authors: Duncan S,Annunziato RA,Dunphy C,LaPointe Rudow D,Shneider BL,Shemesh E

    更新日期:2018-02-01 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


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

    更新日期:2007-11-01 00:00:00

  • Unrelated donor cord blood transplantation for non-malignant disorders in children and adolescents.

    abstract::This study analyzes the data reported to the Korean Cord Blood Registry between 1994 and 2008, involving children and adolescents with non-malignant diseases. Sixty-five patients were evaluated in this study: SAA (n = 24), iBMFS, (n = 16), and primary immune deficiency/inherited metabolic disorder (n = 25). The CI of ...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Park M,Lee YH,Kang HR,Lee JW,Kang HJ,Park KD,Shin HY,Ahn HS,Baek HJ,Kook H,Hwang TJ,Lee JW,Chung NG,Cho B,Kim HK,Lee SH,Yoo KH,Sung KW,Koo HH,Koh KN,Im HJ,Seo JJ,Park JE,Lim YJ,Lyu CJ,Lee JM,Hah JO,K

    更新日期:2014-03-01 00:00:00

  • The challenge of long-term follow-up of survivors of childhood acute leukemia after hematopoietic stem cell transplantation in resource-limited countries: A single-center report from Brazil.

    abstract::With the number of long-term HSCT survivors steadily increasing, attention needs to be focused on the late complications and quality of life. We therefore analyzed the outcome of 101 pediatric patients (<18 years old at the time of HSCT) transplanted for acute leukemia between 1981 and 2015 at Complexo Hospital de Clí...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Marinho DH,Ribeiro LL,Nichele S,Loth G,Koliski A,Mousquer RTG,Funke VAM,Page K,Fasth A,Pasquini R,Boguszewski MCDS,Bonfim C

    更新日期:2020-06-01 00:00:00

  • Growth and weight gain of prepubertal children after cardiac transplantation.

    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


    authors: Peterson RE,Perens GS,Alejos JC,Wetzel GT,Chang RK

    更新日期:2008-06-01 00:00:00

  • Unexpectedly high prevalence of pretransplant abnormal glucose tolerance in pediatric kidney transplant recipients.

    abstract::Several studies suggested that the incidence of new-onset diabetes following pediatric kidney transplantation has increased markedly in recent years, with reported incidence of up to 20%. However, limited information is available regarding the incidence and features of pretransplant status of abnormal glucose toleranc...

    journal_title:Pediatric transplantation

    pub_type: 杂志文章


    authors: Shishido S,Sato H,Asanuma H,Shindo M,Hataya H,Ishikura K,Hamasaki Y,Goto M,Ikeda M,Honda M

    更新日期:2006-02-01 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

  • 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