Transplantation of ex-vivo culture-expanded parental haploidentical mesenchymal stem cells to promote engraftment in pediatric recipients of unrelated donor umbilical cord blood: results of a phase I-II clinical trial.


:Suboptimal neutrophil and platelet recovery after unrelated donor umbilical cord blood transplantation (UCBT) may be due in part to an impaired microenvironment after intensive chemoradiotherapy. In an attempt to speed hematopoietic recovery, 15 pediatric patients with high-risk acute leukemia were enrolled on a single-institution phase I-II clinical trial in which ex-vivo culture-expanded MSCs from haploidentical parental donors were infused at the time of UCBT. Eight patients received MSCs on day 0, with three patients having a second dose infused on day 21. No serious adverse events were observed with any MSC infusion. All eight evaluable patients achieved neutrophil engraftment at a median of 19 days. Probability of platelet engraftment was 75%, at a median of 53 days. With a median follow-up of 6.8 years, five patients remain alive and disease free. The results of this pilot study show that infusion of ex-vivo culture-expanded haploidentical MSCs into unrelated pediatric UCBT recipients can be performed safely. This encouraging safety profile with haploidentical MSCs supports the investigation of unrelated 'off the shelf' allogeneic HLA-mismatched MSC products.


Bone Marrow Transplant


Macmillan ML,Blazar BR,DeFor TE,Wagner JE




Has Abstract


2009-03-01 00:00:00














  • Unrelated cord blood and mismatched unrelated volunteer donor transplants, two alternatives in patients who lack an HLA-identical donor.

    abstract::The aim was to evaluate two transplant strategies for patients who lack HLA-identical donors, namely HLA-A, HLA-B or -DR beta 1 mismatched unrelated donor (MM URD) transplants (n=14) and umbilical cord blood transplants (UCB, n=27). Diagnosis, disease stage and age were similar in the two groups. Cell dose was lower i...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章


    authors: Ringdén O,Okas M,Uhlin M,Uzunel M,Remberger M,Mattsson J

    更新日期:2008-11-01 00:00:00

  • Skin explant culture as a model for cutaneous graft-versus-host disease in humans.

    abstract::An in vitro skin explant model for graft-versus-host disease (GVHD) in humans has been used to study the role of effector T cells in the histological pathogenesis of GVHD. In 11 of 12 experiments clear GVHD changes of grades II-IV were induced in HLA-mismatched skin explants cultured with allogeneic T cells sensitized...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Dickinson AM,Sviland L,Carey P,Reid MM,Hamilton PJ,Pearson AJ,Proctor SJ

    更新日期:1988-07-01 00:00:00

  • Collection of peripheral blood progenitor cells (PBPC) based on a rising WBC and platelet count significantly increases the number of CD34+ cells.

    abstract::The kinetics of mobilization and optimal timing of peripheral blood progenitor cell (PBPC) collection were evaluated in 190 patients with multiple myeloma undergoing stem cell harvest after mobilization with cyclophosphamide, prednisone and G-CSF. There was a strong correlation between the WBC count and the number of ...

    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


    authors: Krieger MS,Schiller G,Berenson JR,Stewart K,Noga SJ,Ballester O,Tarantolo S,Stiff P,Kuhn D,Scherzo E,Sing A,Jacobs C,White JM,DiPersio J

    更新日期:1999-07-01 00:00:00

  • Delayed engraftment and mixed chimerism after HLA-identical sibling donor BMT in Fanconi anaemia.

    abstract::A 12-year-old girl with Fanconi anaemia (FA) received a bone marrow transplant from her HLA-identical brother following conditioning with cyclophosphamide (20 mg/kg), thoraco-abdominal radiation (TAI) (4 Gy) and equine anti-thymocyte globulin (ATG) (90 mg/kg). Engraftment was delayed and initially tenuous, and was fol...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Mital MK,Curtis A,Spencer V,Barge D,Skinner R

    更新日期:1999-07-01 00:00:00

  • Long term renal survival in patients undergoing T-Cell depleted versus conventional hematopoietic stem cell transplants.

    abstract::Calcineurin inhibitor (CNI)-sparing T-cell depleted (TCD) hematopoietic stem cell transplants (HSCTs) are presumed to be less nephrotoxic than conventional HSCTs. We evaluated incidence and risk factors for kidney failure and chronic kidney disease (CKD) in 231 TCD and 212 conventional HSCT recipients. Kidney failure ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Glezerman IG,Devlin S,Maloy M,Bui M,Jaimes EA,Giralt SA,Jakubowski AA

    更新日期:2017-05-01 00:00:00

  • Alefacept treatment for refractory chronic extensive GVHD.

    abstract::Alefacept (Amevive) is an immunosuppressive dimeric fusion protein that is used for psoriasis control. We recently showed its effect in acute steroid-resistant/dependent GVHD. In this study, we describe the effect of alefacept treatment on chronic extensive GVHD (cGVHD). Twelve patients were included in this study; of...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Shapira MY,Abdul-Hai A,Resnick IB,Bitan M,Tsirigotis P,Aker M,Gesundheit B,Slavin S,Or R

    更新日期:2009-02-01 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


    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

  • Phase I trial of histone deacetylase inhibitor panobinostat in addition to glucocorticoids for primary therapy of acute graft-versus-host disease.

    abstract::Glucocorticoids for primary therapy of acute GVHD have limited responses. A phase I/II trial tested 4 weeks of deacetylase inhibitor panobinostat started within 48 h of glucocorticoids (1 mg/kg/day prednisone or equivalent) as primary treatment for patients with either classic acute GVHD (n = 16) or acute GVHD overlap...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Perez L,Fernandez H,Horna P,Riches M,Locke F,Field T,Powers J,Sahakian E,Villagra A,Mishra A,Betts B,Kharfan-Dabaja M,Beato F,Ochoa-Bayona L,Pidala J,Anasetti C

    更新日期:2018-11-01 00:00:00

  • Impaired bone marrow hematopoietic progenitor cell function in rheumatoid arthritis patients candidated to autologous hematopoietic stem cell transplantation.

    abstract::We have evaluated bone marrow morphology, percentage of bone marrow CD34(+) cells, proliferative activity of bone marrow precursors, clonogenic assay (BFU-E and CFU-GM) in short-term bone marrow cultures, and bone marrow cell apoptosis, together with serum TNF-alpha and IL-6, in 16 chronic, refractory RA patients, as ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Porta C,Caporali R,Epis O,Ramaioli I,Invernizzi R,Rovati B,Comolli G,Danova M,Montecucco C

    更新日期:2004-04-01 00:00:00

  • Detection of stromal cells in peripheral blood progenitor cell collections from breast cancer patients.

    abstract::Transplantation of growth factor-mobilized peripheral blood progenitor cells (PBPC) is widely used in the treatment of several neoplastic diseases. While in PBPC harvests the presence of several accessory immune and tumor cells has been documented, that of stromal cells has not been reported. In the present study, we ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Fernández M,Simon V,Herrera G,Cao C,Del Favero H,Minguell JJ

    更新日期:1997-08-01 00:00:00

  • Hematopoietic stem-cell transplantation in children with refractory acute myeloid leukemia.

    abstract::Allogeneic hematopoietic stem cell transplantation (HSCT) can be used to treat children with refractory acute myeloid leukemia (AML). This retrospective analysis aimed to describe the outcomes and risk factors in such children. Data were collected through the nation-wide registry program in Japan. A total of 417 AML (...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章


    authors: Okamoto Y,Kudo K,Tabuchi K,Tomizawa D,Taga T,Goto H,Yabe H,Nakazawa Y,Koh K,Ikegame K,Yoshida N,Uchida N,Watanabe K,Koga Y,Inoue M,Kato K,Atsuta Y,Ishida H

    更新日期:2019-09-01 00:00:00

  • Does haploidentical transplantation in children with primary immunodeficiencies have the potential to exploit donor NK cell alloreactivity?

    abstract::Donor potential to exert NK cell alloreactivity has been shown to confer survival advantage in haploidentical hematopoietic cell transplantation for hematological malignancies. We investigated killer immunoglobulin receptor (KIR) ligand incompatibility in 40 children receiving haploidentical transplantation for primar...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Dal-Cortivo L,Ouachée-Chardin M,Hirsch I,Blanche S,Fischer A,Cavazzana-Calvo M,Caillat-Zucman S

    更新日期:2004-12-01 00:00:00

  • The importance of CD34+/CD33- cells in platelet engraftment after intensive therapy for cancer patients given peripheral blood stem cell rescue.

    abstract::The study was designed to determine whether the number of CD34+/CD33- cells given at autologous peripheral blood stem cell (PBSC) rescue after intensive therapy for cancer was a better predictor of platelet engraftment than the total number of CD34+ cells infused. Comparison between the total number of CD34+ cells/kg ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Millar BC,Millar JL,Shepherd V,Blackwell P,Porter H,Cunningham D,Judson I,Treleaven J,Powles RL,Catovsky D

    更新日期:1998-09-01 00:00:00

  • Correlation of colony-forming cells, long-term culture initiating cells and CD34+ cells in apheresis products from patients mobilized for peripheral blood progenitors with different regimens.

    abstract::Peripheral blood progenitor cell (PBPC) populations used for transplantation were analyzed for the presence of CD34+ cells, colony-forming cells (initial CFC), and long-term culture initiating cells (LTC-IC) cultured on irradiated stroma for 5 weeks. Thirty-eight leukapheresis products were studied from 11 patients wi...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Bender JG,Lum L,Unverzagt KL,Lee W,Van Epps D,George S,Coon J,Ghalie R,McLeod B,Kaizer H

    更新日期:1994-04-01 00:00:00

  • Long-term results of autologous marrow transplantation for relapsed or refractory male or female germ cell tumors.

    abstract::Twenty-one patients with relapsed or refractory germ cell tumors were treated with high-dose chemotherapy and marrow transplantation (HDC/BMT) from 1982-1993. Primary sites of disease were testis (17), ovary (three), and pineal gland (one). Pathology included dysgerminoma (one), choriocarcinoma with adenocarcinoma (on...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章


    authors: Mandanas RA,Saez RA,Epstein RB,Confer DL,Selby GB

    更新日期:1998-03-01 00:00:00

  • Phase-2 trial of an intensified conditioning regimen for allogeneic hematopoietic cell transplant for poor-risk leukemia.

    abstract::Patients with poor-risk leukemia have a high relapse rate despite allogeneic transplant. We report on the phase-2 trial of an intensified allogeneic transplant regimen whose aim was tolerable toxicity and durable remission. Study patients (n=30) had unfavorable first remission cytogenetics, progression from myelodyspl...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Stein AS,O'Donnell MR,Synold TW,Dagis AC,Tsirunyan A,Nademanee AP,Parker PM,Pullarkat VA,Snyder DS,Spielberger RT,Wong JY,Alvarnas JC,Thomas SH,Forman SJ

    更新日期:2011-09-01 00:00:00

  • The changing role of stem cell transplantation in childhood.

    abstract::Over the past decade, relevant improvements and refinements have significantly changed the indications, technique and results obtained with allogeneic hematopoietic SCT (HSCT) in childhood. A fundamental turning point in the history of allogeneic HSCT is represented by the use of placental blood, which was first emplo...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Locatelli F,Giorgiani G,Di-Cesare-Merlone A,Merli P,Sparta V,Moretta F

    更新日期:2008-06-01 00:00:00

  • CR represents an early index of potential long survival in multiple myeloma.

    abstract::To assess the impact of CR on survival in multiple myeloma. Retrospective evaluation of response and survival among 758 consecutive patients with multiple myeloma treated at a single center, of whom 395 patients received intensive therapy supported by autologous stem cells within the first year. Survival times were ca...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Wang M,Delasalle K,Feng L,Thomas S,Giralt S,Qazilbash M,Handy B,Lee JJ,Alexanian R

    更新日期:2010-03-01 00:00:00

  • Horse antilymphocytic globulin in hepatitis B exacerbation after bone marrow transplantation adoptive immunity transfer.

    abstract::We describe the case of a HBsAg+, HBeAg+ carrier, treated with lamivudine, who experienced exacerbation of hepatitis after BMT from an anti-HBs+, anti-HBc+, anti-HBe+ donor. The serological profile of the donor and the timing of exacerbation suggested that the adoptive immunity transfer played a major pathogenetic rol...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Favre C,Menconi MC,Nardi M,Casazza G,Oliveri F,Macchia P,Bonino F,Brunetto MR

    更新日期:2004-05-01 00:00:00

  • A comparison of serological, cellular and DNA-RFLP methods for HLA matching in the selection of related bone marrow donors.

    abstract::Serological, cellular and DNA-RFLP (restriction fragment length polymorphism) methods of determining HLA compatibility between 10 leukaemic patients and potential related bone marrow donors were systematically compared. DR beta/DQ alpha/DQ beta/DNA-RFLP typing of these families gave results in agreement with those obt...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Howell WM,Evans PR,Spellerberg MB,Wilson PJ,Smith JL

    更新日期:1989-01-01 00:00:00

  • Prenatal HLA-matching to determine suitability for allogeneic bone marrow transplantation.

    abstract::For several haematological malignancies, allogeneic stem cell transplantation is the treatment of choice. In most cases an HLA-identical sibling is required. If the mother of a patient is pregnant, cord blood from a related donor, which can be used for stem cell transplantation, might be obtainable in the near future....

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: van den Berg H,Verjaal M,Boer K,Lardy NM

    更新日期:2000-03-01 00:00:00

  • Allogeneic peripheral blood stem cell transplantation using a fludarabine-based low intensity conditioning regimen for malignant lymphoma.

    abstract::Relapse is a serious complication following high-dose therapy and autologous bone marrow transplantation (ABMT) for malignant lymphoma (ML). Allogeneic transplantation (alloSCT) is a therapeutic option. However, it is associated with a high incidence of transplant-related organ toxicity and mortality. We recently repo...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Nagler A,Slavin S,Varadi G,Naparstek E,Samuel S,Or R

    更新日期:2000-05-01 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


    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

  • (90)Y-ibritumomab tiuxetan followed by reduced-intensity conditioning and allo-SCT in patients with advanced follicular lymphoma.

    abstract::Reduced-intensity conditioning (RIC) hematopoietic SCT (HSCT) is a potentially curative therapeutic option for patients with advanced follicular lymphoma (FL), but disease relapse remains the most common cause of failure. Radioimmunoconjugates administered before RIC allo-HSCT may enhance cytoreduction and allow more ...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章


    authors: Abou-Nassar KE,Stevenson KE,Antin JH,McDermott K,Ho VT,Cutler CS,LaCasce AS,Jacobsen ED,Fisher DC,Soiffer RJ,Alyea EP,Koreth J,Freedman AS

    更新日期:2011-12-01 00:00:00

  • Pegfilgrastim compared with filgrastim for cytokine-alone mobilization of autologous haematopoietic stem and progenitor cells.

    abstract::Haematopoietic stem and progenitor cells (HSPC) mobilization, using cytokine-alone, is a well-tolerated regimen with predictable mobilization kinetics. Single-dose pegfilgrastim mobilizes HSPC efficiently; however, there is surprisingly little comparative data on its use without chemotherapy for HSPC mobilization. Peg...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Herbert KE,Gambell P,Link EK,Mouminoglu A,Wall DM,Harrison SJ,Ritchie DS,Seymour JF,Prince HM

    更新日期:2013-03-01 00:00:00

  • High-dose immunosuppressive therapy with PBPC support in the treatment of poor risk multiple sclerosis.

    abstract::High-dose immunoablative chemotherapy with autologous haematopoietic cell support might be beneficial in the treatment of intractable forms of MS. We mobilised PBPC in 11 patients with secondary progressive MS and finally eight patients were grafted after high-dose BEAM chemotherapy with either in vitro or in vivo T c...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章,多中心研究


    authors: Kozák T,Havrdová E,Pit'ha J,Gregora E,Pytlík R,Maaloufová J,Marecková H,Kobylka P,Vodvárková S

    更新日期:2000-03-01 00:00:00

  • Psychological distress in parents consenting to child's bone marrow transplantation.

    abstract::The purpose of this study was to determine the nature and prevalence of the psychological symptomatology in parents of children undergoing bone marrow transplantation (BMT) and to investigate the manner in which certain psychosocial factors are related to parental distress associated with the informed consent process....

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Dermatis H,Lesko LM

    更新日期:1990-12-01 00:00:00

  • Increased plasma level of vascular endothelial glycoprotein thrombomodulin as an early indicator of endothelial damage in bone marrow transplantation.

    abstract::We investigated the nature of hemostatic alterations occurring after bone marrow transplantation. In 45 patients, we evaluated the coagulation parameters, naturally occurring anticoagulants and thrombomodulin at days +15 and +22 after conditioning therapy. It was observed that endothelial cell damage is a central path...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Testa S,Manna A,Porcellini A,Maffi F,Morstabilini G,Denti N,Macchi S,Rosti G,Porcellini G,Cassi D,Ferrari L

    更新日期:1996-08-01 00:00:00

  • Pulmonary toxicity associated with high dose chemotherapy in the treatment of solid tumors with autologous marrow transplant: an analysis of four chemotherapy regimens.

    abstract::We retrospectively reviewed the pulmonary toxicity of six high dose chemotherapy protocols using four chemotherapy regimens in the treatment of solid tumors. All protocols used either high dose cyclophosphamide or ifosfamide in combination with one to three additional chemotherapeutic agents. In each protocol autologo...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Seiden MV,Elias A,Ayash L,Hunt M,Eder JP,Schnipper LE,Frei E 3rd,Antman KH

    更新日期:1992-07-01 00:00:00

  • Low incidence of CMV viremia and disease after allogeneic peripheral blood stem cell transplantation. Role of pretransplant ganciclovir and post-transplant acyclovir.

    abstract::To establish the incidence of CMV viremia after allogeneic blood stem cell transplantation, we studied 51 consecutive allogeneic peripheral blood stem cell (PBSC) transplant recipients. A total of 12 recipients were at moderate risk for CMV disease and 39 were at high risk. Conditioning regimens varied, but GvHD proph...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章


    authors: Verma A,Devine S,Morrow M,Chen YH,Mihalov M,Peace D,Stock W,Pursell K,Wickrema A,Yassine M,Jessop E,van Besien K

    更新日期:2003-05-01 00:00:00