Consensus opinion on immune-mediated cytopenias after hematopoietic cell transplant for inherited metabolic disorders.

Abstract:

:Hematopoietic stem cell transplantation (HCT) has been increasingly used for patients with inherited metabolic disorders (IMD). Immune mediated cytopenias (IMCs) after HCT, manifesting as hemolytic anemia, thrombocytopenia, and/or neutropenia, are recognized as a significant complication in this patient population, yet our understanding of the incidence, risk factors, and pathophysiology is currently limited. Review of the published literature demonstrates a higher incidence in younger patients who undergo HCT for a nonmalignant disease indication. However, a few reports suggest that the incidence is even higher among those with IMD (incidence ranging from 10 to 56%). This review summarizes the literature, provides an approach to better understanding of the possible etiology of IMCs, and proposes a diagnostic and management plan for patients with IMD who develop single or multi-lineage cytopenias after HCT.

journal_name

Bone Marrow Transplant

authors

Gupta AO,Jan Boelens J,Ebens CL,Kurtzberg J,Lund TC,Smith AR,Wagner JE,Wynn R,Blazar BR,Orchard PJ

doi

10.1038/s41409-020-01179-5

subject

Has Abstract

pub_date

2021-01-13 00:00:00

eissn

0268-3369

issn

1476-5365

pii

10.1038/s41409-020-01179-5

pub_type

杂志文章,评审
  • Clonality studies in patients undergoing allogeneic and autologous bone marrow transplantation for haematological malignancies.

    abstract::Twenty three allogeneic bone marrow transplant (BMT) patients with female donors and 23 female autologous transplant patients were assessed for clonality status after transplant to determine the nature of haemopoietic reconstitution. The X chromosome probes PGK, HPRT and M27 beta were used to assess clonality by analy...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Saunders MJ,Jowitt SN,Yin JA

    更新日期:1995-01-01 00:00:00

  • Predictors of non-compliance in autologous hematopoietic SCT patients undergoing out-patient transplants.

    abstract::Non-compliance has received significant attention in medicine, yet few studies have examined its correlates in autologous hematopoietic SCT (AHSCT) patients. This study examined predictors of non-compliance in a sample of 151 AHSCT patients treated in an outpatient setting. Before AHSCT, participants completed a valid...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章

    doi:10.1038/bmt.2011.129

    authors: Mumby PB,Hurley C,Samsi M,Thilges S,Parthasarathy M,Stiff PJ

    更新日期:2012-04-01 00:00:00

  • Evaluation of pretransplant influenza vaccination in hematopoietic SCT: a randomized prospective study.

    abstract::Pretransplant influenza vaccination of the donor or allogeneic hematopoietic SCT (HSCT) candidate was evaluated in a randomized study. One hundred and twenty-two HSCT recipients and their donors were assigned to three randomization groups: no pretransplant vaccination (n=38), donor pretransplant vaccination (n=44) or ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,多中心研究,随机对照试验

    doi:10.1038/bmt.2015.47

    authors: Ambati A,Boas LS,Ljungman P,Testa L,de Oliveira JF,Aoun M,Colturato V,Maeurer M,Machado CM

    更新日期:2015-06-01 00:00:00

  • Bone marrow transplantation in sickle cell disease: the Belgian experience.

    abstract::Twenty eight patients underwent bone marrow transplantation (BMT) for sickle cell anemia in Belgium. The patients were originating from Central Africa, were young and symptomatic. Engraftment occurred in all patients and was sustained in 25. In 3 patients, a bone marrow rejection was observed. One of these patients un...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Vermylen C,Cornu G,Ferster A,Ninane J,Sariban E

    更新日期:1993-01-01 00:00:00

  • The rationale behind autologous autoimmune hematopoietic stem cell transplant conditioning regimens: concerns over the use of total-body irradiation in systemic sclerosis.

    abstract::Hematopoietic stem cell transplantation (HSCT) is becoming an increasingly recognized indication for treatment of autoimmune diseases and severe immune-mediated disorders. However, multicenter registry data have demonstrated higher than anticipated early toxicity, approximately 10% for autoimmune diseases in general, ...

    journal_title:Bone marrow transplantation

    pub_type: 社论,评审

    doi:10.1038/sj.bmt.1704671

    authors: Burt RK,Patel D,Thomas J,Yeager A,Traynor A,Heipe F,Arnold R,Marmont A,Collier D,Glatstein E,Snowden J

    更新日期:2004-11-01 00:00:00

  • Allogeneic haematopoietic stem cell transplantation in children with acute lymphoblastic leukaemia: the BFM/IBFM/EBMT concepts.

    abstract::Children with high risk or relapsed acute lymphoblastic leukaemia (ALL) can benefit from allogeneic haematopoietic stem cell transplantation (SCT). To reduce transplantation-associated complications, the BFM study group, the IBFM study group and the PD-WP-EBMT initiated a prospective cooperative multicentre trail for ...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章,多中心研究

    doi:10.1038/sj.bmt.1704835

    authors: Peters C,Schrauder A,Schrappe M,von Stackelberg A,Stary J,Yaniv I,Gadner H,Klingebiel T,BFM Study Group, the IBFM-Study Group and the Paediatric Disease Working Party of the EBMT.

    更新日期:2005-03-01 00:00:00

  • Incidence and severity of crucial late effects after allogeneic HSCT for malignancy under the age of 3 years: TBI is what really matters.

    abstract::Younger children are considered to be more vulnerable to late effects (LE), which prompted us to study LE in patients after haematopoietic stem cell transplantation (HSCT) for a haematological malignancy before the age of 3. In this multicentre EBMT study, cumulative incidence (CI) and severity of endocrine LE, centra...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,多中心研究

    doi:10.1038/bmt.2016.139

    authors: Bresters D,Lawitschka A,Cugno C,Pötschger U,Dalissier A,Michel G,Vettenranta K,Sundin M,Al-Seraihy A,Faraci M,Sedlacek P,Versluys AB,Jenkins A,Lutz P,Gibson B,Leiper A,Diaz MA,Shaw PJ,Skinner R,O'Brien TA,Salooja

    更新日期:2016-11-01 00:00:00

  • Late cardiovascular morbidity and mortality following pediatric allogeneic hematopoietic cell transplantation.

    abstract::We analyzed late cardiovascular outcomes of 661 patients who survived at least 2 years from hematopoietic cell transplantation for childhood hematologic malignancy between 1995 and 2008. Center for International Blood and Marrow Transplant Research data was supplemented with surveys focused on cardiotoxicity and poten...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/s41409-018-0155-z

    authors: Duncan CN,Brazauskas R,Huang J,Shaw BE,Majhail NS,Savani BN,Flowers MED,Battiwalla M,Beebe K,Dietz AC,Dvorak CC,Giller R,Jacobsohn DA,Kletzel M,Martin PL,Nemecek ER,Nuechterlein B,Talano JA,Pulsipher MA,Baker KS

    更新日期:2018-10-01 00:00:00

  • Three to six year follow-up of normal donors who received recombinant human granulocyte colony-stimulating factor.

    abstract::One hundred and one donors who had received filgrastim (rhG-CSF) for the purpose of donating either granulocytes or peripheral blood stem cells (PBSC) for their relatives more than 3 years ago were contacted. All donors had received daily rhG-CSF at a median dose of 16 microg/kg/day (range 3-16) for a median of 6 days...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1702072

    authors: Cavallaro AM,Lilleby K,Majolino I,Storb R,Appelbaum FR,Rowley SD,Bensinger WI

    更新日期:2000-01-01 00:00:00

  • Cognitive and adaptive behavior 1 and 3 years following bone marrow transplantation.

    abstract::Children receiving a bone marrow transplant (BMT) are at risk for neuropsychological late effects because of potentially neurotoxic chemotherapy and total body irradiation. The goal of this study was to prospectively and longitudinally assess the intellectual and adaptive functioning of children receiving a BMT. This ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1700699

    authors: Kramer JH,Crittenden MR,DeSantes K,Cowan MJ

    更新日期:1997-03-01 00:00:00

  • Multiple cycles of dose-intensive chemotherapy with repeated stem cell support as induction treatment in metastatic breast cancer: a feasibility study.

    abstract::The purpose of this trial was to study feasibility and tolerance of a dose-intensive multicyclic alternating induction chemotherapy with repeated stem cell support in a series of 43 metastatic breast cancer patients. Anthracycline-naive patients (n = 21) received cyclophosphamide 2.5 g/m(2) plus doxorubicin 80 mg/m(2)...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章

    doi:10.1038/sj.bmt.1703125

    authors: García-Rayo S,Pérez-Calvo J,Martín-Algarra S,Martínez-Monge R,Fernández-Hidalgo O,Subirá L,Martínez-Aguillo M,Rebollo J,Azinovic I,Brugarolas A

    更新日期:2001-08-01 00:00:00

  • The role of granulocyte colony-stimulating factor (G-CSF) in the post-transplant period.

    abstract::The administration of G-CSF post transplant has been shown to accelerate the time to neutrophil engraftment. However, this does not necessarily translate into a meaningful clinical benefit to the patient. This randomized study was designed to determine the role of G-CSF following transplantation in patients with breas...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章,多中心研究,随机对照试验

    doi:10.1038/sj.bmt.1703539

    authors: Hornedo J,Solá C,Solano C,López JJ,Alonso S,Lluch A,Ojeda B,Garcia-Conde J,Cortés-Funes H,SOLTI Group.

    更新日期:2002-05-01 00:00:00

  • T-cell depletion: from positive selection to negative depletion in adult patients.

    abstract::Clinical trials have shown that a strategy for haploidentical transplantation based on the infusion of high numbers of T-cell-depleted hematopoietic progenitor cells and no post-transplant immunosuppression controls graft rejection and GvHD in patients with acute leukemia. Overall, event-free survival compares favorab...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2015.88

    authors: Aversa F

    更新日期:2015-06-01 00:00:00

  • Alternative donor SCT for the treatment of MHC class II deficiency.

    abstract::MHC Class II deficiency is a rare primary immunodeficiency disease characterized by absent HLA Class II expression resulting in CD4 lymphopenia, lack of Ag-specific responses and recurrent infection. Without successful allogeneic SCT, most children succumb to infection within the first decade of life. To date, alterna...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2012.140

    authors: Small TN,Qasim W,Friedrich W,Chiesa R,Bleesing JJ,Scurlock A,Veys P,Sparber-Sauer M

    更新日期:2013-02-01 00:00:00

  • Intravenous busulfan for allogeneic hematopoietic stem cell transplantation in infants: clinical and pharmacokinetic results.

    abstract:SUMMARY:High-dose busulfan is an important component of myeloablative regimens. Variable drug exposure may occur following oral administration. Therefore, the use of intravenous busulfan has been advocated. Previous work has suggested a cumulative dosage of 16 mg/kg for haematopoietic transplantation in children less t...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1704209

    authors: Dalle JH,Wall D,Theoret Y,Duval M,Shaw L,Larocque D,Taylor C,Gardiner J,Vachon MF,Champagne MA

    更新日期:2003-10-01 00:00:00

  • Effects of radiation and 4-hydroperoxycyclophosphamide on production of G- and GM-CSF by stromal cells.

    abstract::The effects of in vitro radiation and exposure to 4-hydroperoxycyclophosphamide (4-HC) on the production of G- and GM-CSF by different components of the human hematopoietic microenvironment are described. The marrow microenvironment is composed of fibroblasts, endothelial cells, macrophages, and adipocytes. To study t...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Laver JH,Xu F,Barredo JC,Abboud MR

    更新日期:1992-12-01 00:00:00

  • Relapse after allogeneic bone marrow transplantation for Philadelphia chromosome positive chronic myeloid leukemia: cytogenetic analysis of 24 patients.

    abstract::One hundred patients with chronic myeloid leukemia (CML) submitted to bone marrow transplantation (BMT) were included in a cooperative cytogenetic study. Relapse (defined on the basis of hematological and cytogenetic findings) occurred in 24 (24%) patients at different intervals after BMT. In 18 of these patients (stu...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Zaccaria A,Rosti G,Sessarego M,Frassoni F,Marmont A,Galimberti M,Baronciani D,Filippetti A,Di Bartolomeo P,Di Girolamo G

    更新日期:1988-09-01 00:00:00

  • Feasibility and timing of unrelated donor identification for patients with ALL.

    abstract::Patients with relapsed ALL frequently have short duration second or later remissions, leaving only a brief window of time when it is possible to perform BMT. When no sibling donor is available, identifying and unrelated donor in a timely fashion can be difficult while autologous BMT (ABMT) can be performed more quickl...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Davies SM,Ramsay NK,Weisdorf DJ

    更新日期:1996-05-01 00:00:00

  • Allogeneic marrow transplantation in non-Hodgkin's lymphoma.

    abstract::Nine individuals between 15 and 43 years of age with non-Hodgkin's lymphoma underwent allogeneic marrow transplantation following busulfan 16 mg/kg and cyclophosphamide 120 mg/kg. These individuals were not considered optimal candidates for autologous transplantation chiefly because of marrow involvement or resistance...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章

    doi:

    authors: Copelan EA,Kapoor N,Gibbins B,Tutschka PJ

    更新日期:1990-01-01 00:00:00

  • Effect of autologous hematopoietic stem cell transplantation on multiple sclerosis and neuromyelitis optica spectrum disorder: a PRISMA-compliant meta-analysis.

    abstract::We should consider both the treatment effects and adverse effects of autologous hematopoietic stem cell transplantation (AHSCT) on multiple sclerosis (MS) and neuromyelitis optica spectrum disorders (NMOSD). Articles exploring the effect and safety of AHSCT in the treatment of MS and NMOSD and published before Decembe...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/s41409-020-0810-z

    authors: Zhang P,Liu B

    更新日期:2020-10-01 00:00:00

  • Pilot study of combined cryosupernatant and protein A immunoadsorption exchange in the treatment of grade 3-4 bone marrow transplant-associated thrombotic microangiopathy.

    abstract::Bone marrow transplant-associated thrombotic microangiopathy (BMT-TM) ranges in severity from a self-limiting to a fatal disorder. There is no specific therapy for this condition to date. We have previously described a simple clinical grading system (grade 0-4) for BMT-TM; patients with grade 3-4 BMT-TM do poorly. A p...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Zeigler ZR,Shadduck RK,Nath R,Andrews DF 3rd

    更新日期:1996-01-01 00:00:00

  • Low incidence of hemorrhagic cystitis following ex vivo T-cell depleted haploidentical hematopoietic cell transplantation in children.

    abstract::Hemorrhagic cystitis (HC) is a debilitating complication following allogenic hematopoietic cell transplantation (HCT). HLA disparity and T-cell depletion have been implicated as risk factors for HC. However, reports on the incidence and risk factors for HC in ex vivo T-cell depleted haploidentical HCT (haploHCT) in ch...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/s41409-019-0672-4

    authors: Jepsen C,Turkiewicz D,Ifversen M,Heilmann C,Toporski J,Dykes J,Mellgren K,Pronk CJ

    更新日期:2020-01-01 00:00:00

  • Peripheral blood stem cell for haploidentical transplantation with post-transplant high dose cyclophosphamide: detailed analysis of 181 consecutive patients.

    abstract::While bone marrow (BM) grafts were initially used for T-replete HLA-haploidentical related donors transplantation (Haplo-SCT) with post-transplantation cyclophosphamide (PT-Cy), the use of peripheral blood stem cell (PBSC) remains debated. We thus conducted a detailed analysis evaluating the incidence, risk factors, a...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,多中心研究

    doi:10.1038/s41409-019-0500-x

    authors: Granata A,Fürst S,Bramanti S,Legrand F,Sarina B,Harbi S,De Philippis C,Faucher C,Chabannon C,Lemarie C,Calmels B,Mariotti J,Maisano V,Weiller PJ,Mokart D,Vey N,Bouabdallah R,Castagna L,Blaise D,Devillier R

    更新日期:2019-11-01 00:00:00

  • Blood and marrow transplantation during the emerging COVID-19 pandemic: the Seattle approach.

    abstract::On January 20, 2020, the first patient with coronavirus disease 2019 (COVID-19) in the United States of America was diagnosed in Washington state, which subsequently experienced rapidly increasing numbers of COVID-19 cases, hospitalizations, and deaths. This placed the Seattle Blood and Marrow Transplant Program at Fr...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,评审

    doi:10.1038/s41409-020-01068-x

    authors: Ueda Oshima M,Sandmaier BM,Petersdorf E,Flowers ME,Hill GR,Lee SJ,Appelbaum FR,Carpenter PA,Baker KS,Connelly-Smith L,McCool A,Elgar S,Pergam SA,Liu C,Stewart FM,Mielcarek M

    更新日期:2020-09-26 00:00:00

  • Treatment of acute graft-versus-host disease with PUVA (psoralen and ultraviolet irradiation): results of a pilot study.

    abstract::Acute graft-versus-host disease (aGVHD) is a frequent and major complication after allogeneic stem cell transplantation. For many years psoralen and ultraviolet (UV)-A light have been used in the treatment of chronic cutaneous graft-versus-host disease, but few patients have received PUVA therapy for aGVHD. We assesse...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1701537

    authors: Wiesmann A,Weller A,Lischka G,Klingebiel T,Kanz L,Einsele H

    更新日期:1999-01-01 00:00:00

  • Second EBMT Workshop on reduced intensity allogeneic hemopoietic stem cell transplants (RI-HSCT).

    abstract::A second meeting on reduced intensity allogeneic stem cell transplants (RI-HSCT) was convened in Zurich in February 2001 and focused on transplant-related mortality (TRM) and graft-versus-host disease (GVHD). Retrospective and prospective studies from the EBMT, national groups and single institutions included over 900...

    journal_title:Bone marrow transplantation

    pub_type: 共识发展会议,杂志文章,评审

    doi:10.1038/sj.bmt.1703355

    authors: Bacigalupo A

    更新日期:2002-02-01 00:00:00

  • Comparison of autologous peripheral blood stem cell dosing by ideal vs actual body weight.

    abstract::In this retrospective study, we evaluated the predictability of PBSC dose for hematopoietic engraftment comparing that calculated by ideal body weight (IBW) vs another calculated by actual body weight (ABW) for each patient. Sixty-three consecutive patients treated similarly using one transplant protocol were analyzed...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1701731

    authors: Waples JM,Moreb JS,Sugrue M,Belanger G,Kubilis P,Lynch JW,Gian V,Weeks F,Wingard J

    更新日期:1999-05-01 00:00:00

  • Allogeneic SCT in refractory or relapsed adult ALL is effective without prior reinduction chemotherapy.

    abstract::We present 60 patients with refractory (n=8) or relapsed (n=52) adult ALL who received allogeneic hematopoietic SCT (HSCT) with (n=41) or without (n=19) prior reinduction chemotherapy. In our center, omission of reinduction is recommended if a suitable donor is promptly available, tumor burden is moderate and disease ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2008.258

    authors: Terwey TH,Massenkeil G,Tamm I,Hemmati PG,Neuburger S,Martus P,Dörken B,Hoelzer D,Arnold R

    更新日期:2008-12-01 00:00:00

  • Efficiency of allogeneic hematopoietic SCT from HLA fully-matched non-sibling relatives: a new prospect of exploiting extended family search.

    abstract::The best donors for hematopoietic SCT (HSCT) are fully-matched siblings. In patients without fully-matched siblings, HLA registries or cord blood banks are alternative strategies with some restrictions. Owing to the high rate of consanguineous marriage in our country, between 2006 and 2013, extended family searches we...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2014.307

    authors: Hamidieh AA,Dehaghi MO,Paragomi P,Navaei S,Jalali A,Eslami GG,Behfar M,Ghavamzadeh A

    更新日期:2015-04-01 00:00:00

  • Low CD4 lymphocyte count in a patient with P. carinii pneumonia after autologous bone marrow transplantation.

    abstract::Authors report a case of P. carinii pneumonia in a child with 5% CD4 lymphocytes (absolute number 5/microliters) after autologous bone marrow transplantation followed by pulmonary irradiation. Serial evaluation of CD4 lymphocyte count or percentage or, at least, detection of significant and persistent lymphopenia coul...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Castagnola E,Dini G,Lanino E,Tasso L,Dallorso S,Garaventa A,Rossi GA,Giacchino R

    更新日期:1995-06-01 00:00:00