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De Latour R.P.,Service dHematologie greffe | Porcher R.,Assistance Publique des Hopitaux de Paris | Dalle J.-H.,Service dHematologie pediatrique | Aljurf M.,King Faisal Specialist Hospital And Research Center | And 14 more authors.
Blood | Year: 2013

Although allogeneic hematopoietic stem cell transplantation (HSCT) remains the only curative treatment for patients with Fanconi anemia (FA), published series mostly refer to single-center experience with limited numbers of patients. We analyzed results in 795 patients with FA who underwent first HSCT between May 1972 and January 2010. With a 6-year median follow-up, overall survival was 49% at 20 years (95% confidence interval, 38-65 years). Better outcome was observed for patients transplanted before the age of 10 years, before clonal evolution (ie, myelodysplastic syndrome or acute myeloid leukemia), from a matched family donor, after a conditioning regimen without irradiation, the latter including fludarabine. Chronic graft-versus-host disease and secondary malignancy were deleterious when considered as time-dependent covariates. Age more than 10 years at time of HSCT, clonal evolution as an indication for transplantation, peripheral blood as source of stem cells, and chronic graft-versus-host disease were found to be independently associated with the risk for secondary malignancy. Changes in transplant protocols have significantly improved the outcome of patients with FA, who should be transplanted at a young age, with bone marrow as the source of stem cells. © 2013 by The American Society of Hematology.


Ferrara F.,Stem Cell Transplantation Unit
Expert Opinion on Investigational Drugs | Year: 2012

Introduction: The prognosis of acute myeloid leukemia (AML) is improved in the last two decades, even though induction and consolidation chemotherapy has not involved new drugs. The more effective use of well-known agents as well as refinement of supportive care during the inevitable phase of severe pancytopenia following intensive chemotherapy accounts for the reduction of treatment-related death rate. In addition, mortality due to allogeneic and autologous stem cell transplantation has also been reduced, due to adoption of more effective therapies for graft versus host disease and other transplant-related complications. Areas covered: The multitude of chromosomal and molecular abnormalities makes the treatment of AML a challenging prospect. In addition, genetic aberrations are not mutually exclusive and coexist in the leukemic cells. As a consequence, the clinical development of new biologic agents proceeds slowly. Data for this review were identified from PubMed and references from relevant articles published in English from 2000 to 2011. Expert opinion: In Phase II studies, different new agents have been found to be active in AML and are currently under investigation in Phase III trials also in combination with conventional chemotherapy. In the near future, we would have more information about the possibility of introducing new drugs into daily practice. © 2012 Informa UK, Ltd.


Lawitschka A.,Childrens Cancer Research Institute | Ball L.,Leiden University | Peters C.,Stem Cell Transplantation Unit
Biology of Blood and Marrow Transplantation | Year: 2012

Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative treatment for many children with life-threatening diseases. One of the most significant long-term complications of transplantation is chronic graft-versus-host disease (cGVHD). Although the rates of cGVHD after HSCT are lower in the pediatric population than in adults, cGVHD remains a significant cause of morbidity and mortality. Medicines used to prevent and treat cGVHD remain unsatisfactory, with protracted use of immune suppression necessary and high rates of first-line treatment failure. Efforts to improve salvage treatment are urgently required. Nonpharmacologic strategies attempt to modulate the cellular inflammation response and possibly allow reduction or cessation of immunosuppressive drugs. Mesenchymal stromal cells (MSC) have been shown invitro to mediate a wide variety of immune responses. MSC have been used in the prophylaxis of acute GVHD (aGVHD) and for the treatment of established steroid refractory aGVHD and, more recently, in the management of cGVHD. Extracorporeal photochemotherapy (ECP) has shown promising efficacy in graft-versus-host disease, and may allow a significant reduction in the use of systemic steroids and other immunosuppressants, reducing long-term morbidity and mortality. The accumulated experience shows ECP to be well tolerated, with no clinically significant side effects. © 2012 American Society for Blood and Marrow Transplantation.


Choi I.,National Kyushu Cancer Center | Tanosaki R.,Stem Cell Transplantation Unit | Uike N.,National Kyushu Cancer Center | Utsunomiya A.,Imamura Bun in Hospital | And 5 more authors.
Bone Marrow Transplantation | Year: 2011

We have previously conducted clinical trials of allogeneic hematopoietic SCT with reduced-intensity conditioning regimen (RIC) for adult T-cell leukemia/lymphoma (ATLL) - a disease caused by human T-lymphotropic virus type 1 (HTLV-1) infection and having a dismal prognosis. Long-term follow-up studies of these trials revealed that 10 of the 29 patients have survived for a median of 82 months (range, 54-100 months) after RIC, indicating a possible curability of the disease by RIC. However, we have also observed that the patterns of post-RIC changes in HTLV-1 proviral load over time among the 10 survivors were classified into three patterns. This is the first report to clarify the long-term outcomes after RIC for ATLL patients. © 2011 Macmillan Publishers Limited All rights reserved.


Ferrara F.,Stem Cell Transplantation Unit
Expert Opinion on Pharmacotherapy | Year: 2010

Importance of the field: Acute promyelocytic leukemia (APL) represents a paradigm of therapeutic success in clinical hematology. Since the introduction of all-trans-retinoic-acid in the early 1980s, complete remission rates exceed 90% and the cure rate is > 70%. Notwithstanding, various questions concerning the management of APL remain unanswered. Areas covered in this review: The aim of this article is to focus on still controversial issues in the management of APL, such as the role of arsenic trioxide as front-line therapy, the management of older unfit patients, the potential utility of gemtuzumab-ozogamycin and the effectiveness (if any) of maintenance therapy for patients in molecular remission. In addition, the possibility of reducing the intensity of post-remission therapy, which is associated with substantial morbidity in potentially cured patients, is discussed. What the reader will gain: Current and future therapeutic options for the treatment of newly diagnosed and relapsed APL. Take home message: To date, the therapy of APL is the most successful example of differentiation therapy and its scientific history can serve as a model for subsequent development of similar treatments in other leukemias and cancers. However, treatment strategies continue to evolve rapidly, with particular focus on minimizing the early and late effects of cytotoxic chemotherapy. © 2010 Informa UK Ltd.

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