Mycophenolate mofetil administered every 8 hours in combination with tacrolimus is efficacious in the prophylaxis of acute graft versus host disease in childhood, adolescent, and young adult allogeneic stem cell transplantation recipients.

Abstract:

BACKGROUND:The optimal dose and schedule of mycophenolate mofetil (MMF) in pediatric allogeneic stem cell transplant recipients remains to be determined. We previously reported safety and pharmacokinetics of MMF at 900 mg/m2 q6h dosing. This study was conducted to investigate the efficacy of tacrolimus plus q8h MMF dosing for acute graft versus host disease (GVHD) prophylaxis in a heterogeneous population of children, adolescent, young adult allogeneic stem cell transplant recipients, utilizing multiple allogeneic donor sources. PROCEDURE:GVHD prophylaxis consisted of tacrolimus 0.03-0.04 mg/kg/day intravenous continuous infusion or 0.12-0.16 mg/kg/day orally divided q8-12h and MMF 900 mg/m2 /dose (max. 1.5 g) or 15 mg/kg/dose intravenous/orally (age ≥18 years) q8h starting on Day +1. MMF was discontinued on Day +30 or Day +60 in the absence of acute GVHD. Thirty-five children, adolescents, and young adult allogeneic stem cell transplant recipients with malignant and nonmalignant disorders were enrolled. RESULTS:Kaplan-Meier probability of grade II-IV and grade III-IV acute GVHD was 22.8% (CI95 : 5.2-47.9 [where CI stands for confidence interval]) and 5.7% (CI95 : 0-48.9), respectively. Probability of extensive and limited chronic GVHD was 22.6% (CI95 : 3.4-52.2) and 12.2% (CI95 : 0.3-45.7), respectively. Probability of 1 year overall survival was 82% (CI95 : 64.1-99.8). Myeloablative conditioning was predictive of higher risk of acute GVHD in the univariate analysis (P = 0.01, hazard ratio = 6.6, CI95 : 0.91-48). CONCLUSION:This study demonstrated a low probability of acute and chronic GVHD in a diverse cohort of childhood, adolescent, and young adult allogeneic stem cell transplant recipients following MMF q8h plus tacrolimus prophylaxis.

journal_name

Pediatr Blood Cancer

journal_title

Pediatric blood & cancer

authors

Militano O,Ozkaynak MF,Mehta B,van deVen C,Hamby C,Cairo MS

doi

10.1002/pbc.27091

subject

Has Abstract

pub_date

2018-08-01 00:00:00

pages

e27091

issue

8

eissn

1545-5009

issn

1545-5017

journal_volume

65

pub_type

临床试验,杂志文章
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    更新日期:2009-09-01 00:00:00

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    doi:10.1002/pbc.24284

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    更新日期:2018-11-01 00:00:00

  • Relapse after localized rhabdomyosarcoma: Evaluation of the efficacy of second-line chemotherapy.

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    journal_title:Pediatric blood & cancer

    pub_type: 临床试验,杂志文章,多中心研究

    doi:10.1002/pbc.25622

    authors: Winter S,Fasola S,Brisse H,Mosseri V,Orbach D

    更新日期:2015-11-01 00:00:00

  • The role of the initial bone marrow aspirate in the diagnosis of hemophagocytic lymphohistiocytosis.

    abstract:BACKGROUND:The identification of hemophagocytosis (HPC) in tissue or bone marrow (BM) represents only one of 5/8 criteria needed for the diagnosis of hemophagocytic lymphohistiocytosis (HLH). Yet, confirmation of HPC in bone marrow aspirates (BMA) is often relied upon to make therapeutic decisions. There is no standard...

    journal_title:Pediatric blood & cancer

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    doi:10.1002/pbc.21564

    authors: Gupta A,Tyrrell P,Valani R,Benseler S,Weitzman S,Abdelhaleem M

    更新日期:2008-09-01 00:00:00

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    abstract::The Standards, Options, and Recommendations (SOR) project undertaken by the French National Federation of Cancer Centers (FNCLCC) to develop and disseminate clinical practice guidelines in oncology has now been taken over by the French National Cancer Institute. In 2007, the SOR updated the information related to the ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,评审

    doi:10.1002/pbc.21953

    authors: Marec-Berard P,Chastagner P,Kassab-Chahmi D,Casadevall N,Marchal C,Misset JL,Ray-Coquard I

    更新日期:2009-07-01 00:00:00

  • Treatment of pharyngeal non-Hodgkin lymphoma in a patient with Wiskott-Aldrich syndrome.

    abstract::Wiskott-Aldrich syndrome (WAS) is characterized by primary immunodeficiency, thrombocytopenia and eczema. Patients with WAS have an increased risk to develop tumors. Non-Hodgkin lymphoma (NHL) represents the most common malignancy occurring in WAS-affected patients, diffuse-large-B-cell lymphoma is the most frequently...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.23393

    authors: Coccia P,Mastrangelo S,Ruggiero A,Scalzone M,Rosolen A,Maurizi P,Riccardi R

    更新日期:2012-08-01 00:00:00

  • Circulating tumor DNA in neuroblastoma.

    abstract::As a sympathetic nervous system-derived tumor, aggressive neuroblastoma (NB) is currently attracting interest from researchers seeking diagnostic and prognostic markers via less invasive procedures. The analysis of circulating tumor DNA (ctDNA) in peripheral blood can provide genetic information from multiple tumor le...

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    pub_type: 杂志文章,评审

    doi:10.1002/pbc.28311

    authors: Wei M,Ye M,Dong K,Dong R

    更新日期:2020-10-01 00:00:00

  • Hepatoblastoma in patients with molecularly proven familial adenomatous polyposis: Clinical characteristics and rationale for surveillance screening.

    abstract::Familial adenomatous polyposis (FAP) due to APC mutation is associated with an increased risk of hepatoblastoma. All cases of hepatoblastoma in patients with FAP reported in the literature were reviewed. One hundred and nine patients were identified. Thirty-five patients (of 49 with data) were diagnosed with hepatobla...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,评审

    doi:10.1002/pbc.27103

    authors: Trobaugh-Lotrario AD,López-Terrada D,Li P,Feusner JH

    更新日期:2018-08-01 00:00:00

  • Preventing Infections in Sickle Cell Disease: The Unfinished Business.

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    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,评审

    doi:10.1002/pbc.25911

    authors: Obaro SK,Iroh Tam PY

    更新日期:2016-05-01 00:00:00

  • Risk prediction of fever in neutropenia in children with cancer: a step towards individually tailored supportive therapy?

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    doi:10.1002/pbc.21726

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    更新日期:2008-12-01 00:00:00

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    pub_type: 杂志文章,评审

    doi:10.1002/pbc.24116

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    更新日期:2012-07-01 00:00:00

  • Ewing sarcoma/primitive neuroectodermal tumor of the kidney in a child.

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    pub_type: 杂志文章

    doi:10.1002/pbc.20831

    authors: Maeda M,Tsuda A,Yamanishi S,Uchikoba Y,Fukunaga Y,Okita H,Hata J

    更新日期:2008-01-01 00:00:00

  • Treatment of the X-linked lymphoproliferative, Griscelli and Chédiak-Higashi syndromes by HLH directed therapy.

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    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.21790

    authors: Trottestam H,Beutel K,Meeths M,Carlsen N,Heilmann C,Pasić S,Webb D,Hasle H,Henter JI

    更新日期:2009-02-01 00:00:00

  • A novel homozygous VPS45 p.P468L mutation leading to severe congenital neutropenia with myelofibrosis.

    abstract::VPS45-associated severe congenital neutropenia (SCN) is a rare disorder characterized by life-threating infections, neutropenia, neutrophil and platelet dysfunction, poor response to filgrastim, and myelofibrosis with extramedullary hematopoiesis. We present a patient with SCN due to a homozygous c.1403C>T (p.P468L) m...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.26571

    authors: Shah RK,Munson M,Wierenga KJ,Pokala HR,Newburger PE,Crawford D

    更新日期:2017-09-01 00:00:00

  • Rubinstein-Taybi syndrome predisposing to non-WNT, non-SHH, group 3 medulloblastoma.

    abstract::Medulloblastomas (MB) are classified in four subgroups: the well defined WNT and Sonic Hedgehog (SHH) subgroups, and the less defined groups 3 and 4. They occasionally occur in the context of a cancer predisposition syndrome. While germline APC mutations predispose to WNT MB, germline mutations in SUFU, PTCH1, and TP5...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.24765

    authors: Bourdeaut F,Miquel C,Richer W,Grill J,Zerah M,Grison C,Pierron G,Amiel J,Krucker C,Radvanyi F,Brugieres L,Delattre O

    更新日期:2014-02-01 00:00:00

  • Histology, fusion status, and outcome in metastatic rhabdomyosarcoma: A report from the Children's Oncology Group.

    abstract:BACKGROUND:Distinguishing alveolar rhabdomyosarcoma (ARMS) from embryonal rhabdomyosarcoma (ERMS) has historically been of prognostic and therapeutic importance. However, classification has been complicated by shifting histologic criteria required for an ARMS diagnosis. Children's Oncology Group (COG) studies after IRS...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.26645

    authors: Rudzinski ER,Anderson JR,Chi YY,Gastier-Foster JM,Astbury C,Barr FG,Skapek SX,Hawkins DS,Weigel BJ,Pappo A,Meyer WH,Arnold MA,Teot LA,Parham DM

    更新日期:2017-12-01 00:00:00

  • Adherence to prompt fever evaluation in children with sickle cell disease and the health belief model.

    abstract:BACKGROUND:Children with sickle cell disease (SCD) are at increased risk of death from invasive bacterial infections. Emergent evaluation of fever allows early treatment of potentially fatal infections. Limited data exist regarding caregiver adherence to physician recommendations of prompt medical evaluation of fever i...

    journal_title:Pediatric blood & cancer

    pub_type: 临床试验,杂志文章

    doi:10.1002/pbc.25634

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    更新日期:2015-11-01 00:00:00

  • Neuropsychological outcomes of a randomized trial of prednisone versus dexamethasone in acute lymphoblastic leukemia: findings from Dana-Farber Cancer Institute All Consortium Protocol 00-01.

    abstract:BACKGROUND:Dexamethasone is more efficacious than prednisone in the treatment of acute lymphoblastic leukemia (ALL), but has also been associated with greater toxicity. We compared neuropsychological outcomes for patients treated on DFCI ALL Consortium Protocol 00-01, which included a randomized comparison of the two s...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,随机对照试验

    doi:10.1002/pbc.24666

    authors: Waber DP,McCabe M,Sebree M,Forbes PW,Adams H,Alyman C,Sands SA,Robaey P,Romero I,Routhier MÈ,Girard JM,Sallan SE,Silverman LB

    更新日期:2013-11-01 00:00:00

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    abstract:BACKGROUND:Sickle cell disease (SCD) is an autosomal recessive hemoglobinopathy associated with morbidity and mortality. We sought to understand family planning intentions of parents of young children with SCD including the awareness of three reproductive options (adoption, in vitro fertilization with egg/sperm donatio...

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    pub_type: 杂志文章

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    更新日期:2020-05-01 00:00:00

  • Opioid selection during sickle cell pain crisis and its impact on the development of acute chest syndrome.

    abstract:BACKGROUND:The hallmark of sickle cell disease (SCD) is recurrent, painful vaso-occlusive episodes (VOC) and is the most common reason for hospitalization in SCD patients. Narcotics, particularly morphine, along with fluid hydration are standard treatments for painful episodes but have been associated with the developm...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.20403

    authors: Buchanan ID,Woodward M,Reed GW

    更新日期:2005-10-15 00:00:00

  • Evaluation of partial and total splenectomy in children with sickle cell disease using an Internet-based registry.

    abstract:BACKGROUND:Clinical outcomes of children with sickle cell disease (SCD) who undergo total or partial splenectomy (PS) are poorly defined. The purpose of this retrospective study was to initiate an Internet-based registry to facilitate analysis of clinical outcomes for these children. We hypothesized that both surgical ...

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    pub_type: 临床试验,杂志文章

    doi:10.1002/pbc.24057

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    更新日期:2012-07-15 00:00:00

  • Clinical phenotype and genetic analysis of RPS19, RPL5, and RPL11 genes in Greek patients with Diamond Blackfan Anemia.

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    pub_type: 杂志文章

    doi:10.1002/pbc.20739

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    更新日期:2006-11-01 00:00:00

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    更新日期:2019-07-01 00:00:00

  • Pediatric residents' perceived barriers to opioid use in sickle cell disease pain management.

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    pub_type: 杂志文章

    doi:10.1002/pbc.27535

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    更新日期:2019-02-01 00:00:00

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    doi:10.1002/pbc.26929

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    更新日期:2018-04-01 00:00:00

  • The pediatric hydroxyurea phase III clinical trial (BABY HUG): challenges of study design.

    abstract::Evidence of the laboratory benefits of hydroxyurea and its clinical efficacy in reducing acute vaso-occlusive events in adults and children with sickle cell anemia has accumulated for more than 15 years. A definitive clinical trial showing that hydroxyurea can also prevent organ damage might support widespread use of ...

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    更新日期:2010-02-01 00:00:00