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Neunert C.,Columbia University | Farah R.,Saint George Hospital University Medical Center | Yacobovich J.,Tel Aviv University | Neufeld E.,Dana Farber Boston Childrens Center for Cancer and Blood Disorders
Seminars in Hematology | Year: 2016

A recent Intercontinental Cooperative ITP Study Group (ICIS) meeting in September 2015 focused on immunomodulation across the spectrum of autoimmune conditions. It became clear to the attendees that in this wide range of conditions, there is a subset of patients that remain highly refractory to first line therapy. Therapeutic approaches to these patients vary greatly and while many different immunomodulatory agents have been investigated, few have seen universal success. We outline here the landscape of immunomodulation therapy for refractory patients across a variety of autoimmune conditions in order to highlight the variety of agents that have been studied, the lack of overall consensus about management, and the need for ongoing research in this area. © 2016 Elsevier Inc.. Source


Cummings C.L.,Harvard University | Geis G.M.,Tufts University | Kesselheim J.C.,Dana Farber Boston Childrens Center for Cancer and Blood Disorders | Sayeed S.,Harvard University
Journal of Perinatology | Year: 2015

Objective: The objectives of this study were to determine the perceived adequacy of ethics and professionalism education for neonatal-perinatal fellows in the United States, and to measure confidence of fellows and recent graduates when navigating ethical issues. Study Design: Neonatal-Perinatal Fellowship Directors, fellows and recent graduates were surveyed regarding the quality and type of such education during training, and perceived confidence of fellows/graduates in confronting ethical dilemmas. Result: Forty-six of 97 Directors (47%) and 82 of 444 fellows/graduates (18%) completed the surveys. Over 97% of respondents agreed that ethics training is 'important/very important'. Only 63% of Directors and 37% of fellows/graduates rated ethics education as 'excellent/very good' (P=0.004). While 96% of Directors reported teaching of ethics, only 70% of fellows/graduates reported such teaching (P<0.001). Teaching methods and their perceived effectiveness varied widely. Conclusion: Training in ethics and professionalism for fellows is important, yet currently insufficient; a more standardized curriculum may be beneficial to ensure that trainees achieve competency. © 2015 Nature America, Inc. All rights reserved. Source


Barr R.D.,McMaster University | Baez F.,Hospital de Manuel de Jesus Rivera La Mascota | Bonilla M.,Benjamin Bloom Hospital | Moreno B.,Hospital del Nino | And 14 more authors.
Pediatric Blood and Cancer | Year: 2014

Bridging the survival gap for children with cancer, between those (the great majority) in low and middle income countries (LMIC) and their economically advantaged counterparts, is a challenge that has been addressed by twinning institutions in high income countries with centers in LMIC. The long-established partnership between a Central American consortium-Asociación de Hemato-Oncología Pediátrica de Centro América (AHOPCA)-and institutions in Europe and North America provides a striking example of such a twinning program. The demonstrable success of this endeavor offers a model for improving the health outcomes of children with cancer worldwide. As this remarkable enterprise celebrates its 15th anniversary, it is appropriate to reflect on its origin, subsequent growth and development, and the lessons it provides for others embarking on or already engaged in similar journeys. Many challenges have been encountered and not all yet overcome. Commitment to the endeavor, collaboration in its achievements and determination to overcome obstacles collectively are the hallmarks that stamp AHOPCA as a particularly successful partnership in advancing pediatric oncology in the developing world. © 2013 Wiley Periodicals, Inc. Source


Neufeld E.J.,Dana Farber Boston Childrens Center for Cancer and Blood Disorders | Negrier C.,University of Lyon | Arkhammar P.,Novo Nordisk AS | Benchikh el Fegoun S.,Novo Nordisk AS | And 3 more authors.
Blood Reviews | Year: 2015

This updated safety review summarises the large body of safety data available on the use of recombinant activated factor VII (rFVIIa) in approved indications: haemophilia with inhibitors, congenital factor VII (FVII) deficiency, acquired haemophilia and Glanzmann's thrombasthenia. Accumulated data up to 31 December 2013 from clinical trials as well as post-marketing data (registries, literature reports and spontaneous reports) were included. Overall, rFVIIa has shown a consistently favourable safety profile, with no unexpected safety concerns, in all approved indications. No confirmed cases of neutralising antibodies against rFVIIa have been reported in patients with congenital haemophilia, acquired haemophilia or Glanzmann's thrombasthenia. The favourable safety profile of rFVIIa can be attributed to the recombinant nature of rFVIIa and its localised mechanism of action at the site of vascular injury. Recombinant FVIIa activates factor X directly on the surface of activated platelets, which are present only at the site of injury, meaning that systemic activation of coagulation is avoided and the risk of thrombotic events (TEs) thus reduced. Nonetheless, close monitoring for signs and symptoms of TE is warranted in all patients treated with any pro-haemostatic agent, including rFVIIa, especially the elderly and any other patients with concomitant conditions and/or predisposing risk factors to thrombosis. © 2015 Elsevier Ltd. Source


Wang L.D.,Joslin Diabetes Center | Wang L.D.,Harvard Stem Cell Institute | Wang L.D.,Harvard University | Wang L.D.,Dana Farber Boston Childrens Center for Cancer and Blood Disorders | And 35 more authors.
Leukemia | Year: 2015

Mast cells (MCs) are critical components of the innate immune system and important for host defense, allergy, autoimmunity, tissue regeneration and tumor progression. Dysregulated MC development leads to systemic mastocytosis (SM), a clinically variable but often devastating family of hematologic disorders. Here we report that induced expression of Lin28, a heterochronic gene and pluripotency factor implicated in driving a fetal hematopoietic program, caused MC accumulation in adult mice in target organs such as the skin and peritoneal cavity. In vitro assays revealed a skewing of myeloid commitment in LIN28B-expressing hematopoietic progenitors, with increased levels of LIN28B in common myeloid and basophil-MC progenitors altering gene expression patterns to favor cell fate choices that enhanced MC specification. In addition, LIN28B-induced MCs appeared phenotypically and functionally immature, and in vitro assays suggested a slowing of MC terminal differentiation in the context of LIN28B upregulation. Finally, interrogation of human MC leukemia samples revealed upregulation of LIN28B in abnormal MCs from patients with SM. This work identifies Lin28 as a novel regulator of innate immune function and a new protein of interest in MC disease. © 2015 Macmillan Publishers Limited. Source

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