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Seminars in Hematology

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https://www.readbyqxmd.com/read/29958565/when-there-is-no-match-the-game-is-not-over-alternative-donor-options-for-hematopoietic-stem-cell-transplantation-in-sickle-cell-disease
#1
REVIEW
Jacinth J Joseph, Allistair A Abraham, Courtney D Fitzhugh
Many patients with sickle cell disease experience severe morbidity and early mortality. The only curative option remains hematopoietic stem cell transplantation. Although HLA-matched sibling transplantation has been very successful for adults and children, the vast majority of patients with sickle cell disease do not have an HLA-matched sibling. Alternative donor options include haploidentical, unrelated umbilical cord blood, and matched unrelated donor transplantation. This report summarizes major alternative donor transplantation studies reported to date and ongoing and upcoming clinical trials...
April 2018: Seminars in Hematology
https://www.readbyqxmd.com/read/29958564/curative-therapies-allogeneic-hematopoietic-cell-transplantation-from-matched-related-donors-using-myeloablative-reduced-intensity-and-nonmyeloablative-conditioning-in-sickle-cell-disease
#2
REVIEW
Gregory M T Guilcher, Tony H Truong, Santosh L Saraf, Jacinth J Joseph, Damiano Rondelli, Matthew M Hsieh
Sickle cell disease (SCD) chronically damages multiple organs over the lifetime of affected individuals. Allogeneic hematopoietic cell transplantation (allo-HCT) is the most studied curative intervention. Fully matched related marrow, peripheral blood derived, or cord blood HCT have the best transplant outcome for symptomatic patients with SCD. For patients with asymptomatic or milder disease who have this donor option available, risks and benefits of HCT should be discussed among the patient, family, treating hematologist, and transplant physician, and decision to proceed to HCT should be individualized...
April 2018: Seminars in Hematology
https://www.readbyqxmd.com/read/29958563/genetic-therapies-for-sickle-cell-disease
#3
REVIEW
Erica B Esrick, Daniel E Bauer
After decades with few novel therapeutic options for sickle cell disease (SCD), autologous hematopoietic stem cell (HSC) based genetic therapies including lentiviral gene therapy (GT), and genome editing (GE) now appear imminent. Lentiviral GT has advanced considerably in the past decade with promising clinical trial results in multiple disorders. For β-hemoglobinopathies, GT strategies of gene addition and fetal hemoglobin induction through BCL11A regulation are both being evaluated in open clinical trials...
April 2018: Seminars in Hematology
https://www.readbyqxmd.com/read/29958562/fetal-hemoglobin-induction-by-epigenetic-drugs
#4
REVIEW
Donald Lavelle, James Douglas Engel, Yogen Saunthararajah
Fetal hemoglobin (HbF) inhibits the root cause of sickle pathophysiology, sickle hemoglobin polymerization. Individuals who naturally express high levels of HbF beyond infancy thus receive some protection from sickle complications. To mimic this natural genetic experiment using drugs, one guiding observation was that HbF is increased during recovery of bone marrow from extreme stress. This led to evaluation and approval of the cytotoxic (cell killing) drug hydroxyurea to treat sickle cell disease. Cytotoxic approaches are limited in potency and sustainability, however, since they require hematopoietic reserves sufficient to repeatedly mount recoveries from stress that destroys their counterparts, and such reserves are finite...
April 2018: Seminars in Hematology
https://www.readbyqxmd.com/read/29958561/sickle-cell-disease-unanswered-questions-and-future-directions-in-therapy
#5
EDITORIAL
Swee Lay Thein, John Tisdale
No abstract text is available yet for this article.
April 2018: Seminars in Hematology
https://www.readbyqxmd.com/read/29759154/mrd-testing-in-multiple-myeloma-the-main-future-driver-for-modern-tailored-treatment
#6
REVIEW
Ola Landgren, Sydney X Lu, Malin Hultcrantz
The past decade, several highly efficacious drugs have been approved for the treatment of multiple myeloma. Many of these newer drugs are less toxic than older chemotherapy drugs. Using modern combination therapy in newly diagnosed multiple myeloma patients, high proportions of newly diagnosed multiple myeloma patients obtain minimal residual disease (MRD) negativity and MRD testing has rapidly become an integral part of clinical trials focusing on patients in this setting. Only recently, MRD negativity was reported in clinical trials focusing on older newly diagnosed multiple myeloma patients (ie, nontransplant candidates), as well as studies focusing on patients with relapsed or refractory multiple myeloma...
January 2018: Seminars in Hematology
https://www.readbyqxmd.com/read/29759153/mass-spectrometry-methods-for-detecting-monoclonal-immunoglobulins-in-multiple-myeloma-minimal-residual-disease
#7
REVIEW
Katie L Thoren
Mass spectrometry methods that can detect low levels of monoclonal immunoglobulin in serum have recently been developed. These assays are based on the principle that each immunoglobulin has a unique amino acid sequence and therefore, has a unique mass. This mass can be used as a surrogate marker in order to monitor a patient's disease over time and at low levels. Here, we explain these methods, discuss their advantages and disadvantages and how they may be used to monitor monoclonal immunoglobulins for minimal residual disease detection in multiple myeloma...
January 2018: Seminars in Hematology
https://www.readbyqxmd.com/read/29759152/minimal-residual-disease-detection-by-flow-cytometry-in-multiple-myeloma-why-and-how
#8
REVIEW
Mikhail Roshal
The outlook for myeloma patients has steadily improved with the introduction of newer drug combinations in recent years. Unlike older therapies that largely achieved only modest levels of neoplastic clone reduction, the newer drug combinations have led to deeper suppression of myeloma clones in most patients. Frequently the neoplastic clones become undetectable with traditional disease evaluation approaches. Recent studies using ultrasensitive disease monitoring have demonstrated that patients with disease undetectable by traditional techniques show wide heterogeneity in disease levels varying by several orders of magnitude...
January 2018: Seminars in Hematology
https://www.readbyqxmd.com/read/29759151/circulating-tumour-dna-for-detecting-minimal-residual-disease-in-multiple-myeloma
#9
REVIEW
Trevor J Pugh
Circulating tumor DNA faithfully recapitulates somatic mutations detected in bone marrow aspirates from patients with newly diagnosed or relapsed or recurrent myeloma. Extending these methods to enable detection of minimal residual disease will require increased sensitivity and breadth of genomic assays to maximize information content from small quantities of cell-free DNA; as well as definition of a clinically meaningful ctDNA concentration in comparison with conventional bone marrow cell-count thresholds...
January 2018: Seminars in Hematology
https://www.readbyqxmd.com/read/29759150/comprehensive-characterization-of-circulating-and-bone-marrow-derived-multiple-myeloma-cells-at-minimal-residual-disease
#10
REVIEW
Johannes M Waldschmidt, Praveen Anand, Birgit Knoechel, Jens G Lohr
The presence or absence of minimal residual disease (MRD) in patients with multiple myeloma (MM) has emerged as a useful marker to determine the depth of remission. MRD negativity as an endpoint has been shown to be associated with improved progression-free survival in many studies. MRD detection is therefore part of numerous clinical trial protocols for MM. At the present time, two methodologies are most widely accepted for MRD detection: (1) multicolor flow cytometry and (2) next-generation sequencing-based clonotype detection...
January 2018: Seminars in Hematology
https://www.readbyqxmd.com/read/29759149/functional-imaging-methods-for-assessment-of-minimal-residual-disease-in-multiple-myeloma-current-status-and-novel-immunopet-based-methods
#11
REVIEW
Neeta Pandit-Taskar
Imaging plays a key role in assessment of myeloma. Osteolytic bone lesions are optimally assessed using structural imaging, however the structural changes lag the functional changes in the disease. Functional imaging with fluoro deoxy glucose (FDG) positron emission tomography (PET) computerized tomography (CT) is useful in assessment of high-risk myeloma. FDG PET provides prognostic information and is helpful in monitoring response to therapy. However, it is nonspecific and may not be optimal in assessing treatment response to immunotherapeutic agents...
January 2018: Seminars in Hematology
https://www.readbyqxmd.com/read/29759148/minimal-residual-disease-in-multiple-myeloma-use-of-magnetic-resonance-imaging
#12
REVIEW
Jens Hillengass, Maximilian Merz, Stefan Delorme
The increasing percentage of patients achieving deep responses in multiple myeloma has led to the need for more sophisticated instruments to measure residual disease as a potential source of relapse. As minimal residual disease assessment is mostly performed on a bone marrow specimen from a certain area of the body, such samples have the limitation that they might not really represent the actual tumor burden, because focal accumulations of malignant cells might be either hit or missed. Magnetic resonance imaging is a highly sensitive technique for the assessment of tumor burden and can be performed as whole-body protocol, overcoming the problem of sampling error for minimal residual disease assessment...
January 2018: Seminars in Hematology
https://www.readbyqxmd.com/read/29759147/minimal-residual-disease-detection-of-myeloma-using-sequencing-of-immunoglobulin-heavy-chain-gene-vdj-regions
#13
REVIEW
Caleb Ho, Maria E Arcila
After therapy or stem cell transplantation, multiple myeloma patients achieving complete response or stringent complete response can still have a significant risk of disease relapse. This highlights the importance of using highly sensitive laboratory methods for minimal residual disease detection and prognostication. Older methods such as allele-specific oligonucleotide real time quantitative polymerase chain reaction and fluorescent polymerase chain reaction have their drawbacks. Meanwhile, the recent generation of multiparametric flow cytometry and next-generation sequencing (NGS)-based detection methods currently offer the highest technical sensitivities, and are likely to gain more widespread use and be recognized as the standard of care for disease monitoring in myeloma patients...
January 2018: Seminars in Hematology
https://www.readbyqxmd.com/read/29759146/mrd-testing-in-multiple-myeloma-from-a-surrogate-marker-of-clinical-outcomes-to-an-every-day-clinical-tool
#14
EDITORIAL
Ola Landgren
Minimal residual disease (MRD) testing in multiple myeloma is here to stay. Studies show that MRD negativity is consistently associated with longer progression-free survival (PFS). It is just a matter of time until MRD negativity will become a regulatory endpoint for drug approval. Until that can happen, more analysis will be required to define the exact details of MRD in the regulatory setting. For example, for randomized studies there is need to define the amount of improvement in MRD negativity between the experimental arm and the control arm at a given time-point for a drug to obtain regulatory accelerated approval...
January 2018: Seminars in Hematology
https://www.readbyqxmd.com/read/29153081/risks-and-benefits-of-twitter-use-by-hematologists-oncologists-in-the-era-of-digital-medicine
#15
REVIEW
Deanna J Attai, Patricia F Anderson, Michael J Fisch, David L Graham, Matthew S Katz, Jennifer Kesselheim, Merry Jennifer Markham, Nathan A Pennell, Mina S Sedrak, Michael A Thompson, Audun Utengen, Don S Dizon
Twitter use by physicians, including those in the hematology-oncology field, is increasing. This microblogging platform provides a means to communicate and collaborate on a global scale. For the oncology professional, an active Twitter presence provides opportunities for continuing medical education, patient engagement and education, personal branding, and reputation management. However, because Twitter is an open, public forum, potential risks such as patient privacy violations, personal information disclosures, professionalism lapses, and time management need to be considered and managed...
October 2017: Seminars in Hematology
https://www.readbyqxmd.com/read/29153080/impact-of-social-media-for-the-hematologist-oncologist
#16
REVIEW
Nour Abuhadra, Navneet S Majhail, Aziz Nazha
In the era of modern communication, the physician and patient relationship has evolved to include an entirely new dimension-social media. This new dimension offers several opportunities for patient education, research and its dissemination, and professional development for health care providers; it can also serve as a platform for addressing important public health issues. However, these advantages come with challenges such as threats to patient and professional privacy. In this article, we dissect the benefits and drawbacks of this social evolution on the practicing hematologist-oncologist...
October 2017: Seminars in Hematology
https://www.readbyqxmd.com/read/29153079/disease-specific-hashtags-and-the-creation-of-twitter-medical-communities-in-hematology-and-oncology
#17
REVIEW
Naveen Pemmaraju, Michael A Thompson, Muzaffar Qazilbash
Twitter is being increasingly used for information gathering and dissemination of ideas in both medical practice and scientific research. A major limitation to its use has been the surplus of available information and difficulty in categorizing that information into topics of individual interest. However, a Twitter feature known as the hashtag (#), which denotes a specific category or topic, helps in streamlining this wealth of information. The creation and adoption of disease-specific hashtags by healthcare stakeholders has led to a greater uniformity of medical discussions that can be retrieved and referenced at later time-points...
October 2017: Seminars in Hematology
https://www.readbyqxmd.com/read/29153078/the-use-and-impact-of-twitter-at-medical-conferences-best-practices-and-twitter-etiquette
#18
REVIEW
Naveen Pemmaraju, Ruben A Mesa, Navneet S Majhail, Michael A Thompson
The use of social media, and in particular, Twitter, for professional use among healthcare providers is rapidly increasing across the world. One medical subspecialty that is leading the integration of this new platform for communication into daily practice and for information dissemination to the general public is the field of hematology/oncology. A growing amount of research in this area demonstrates that there is increasing interest among physicians to learn not only how to use social media for consumption of educational material, but also how to generate and contribute original content in one's interest/expert areas...
October 2017: Seminars in Hematology
https://www.readbyqxmd.com/read/29153077/twitter-101-and-beyond-introduction-to-social-media-platforms-available-to-practicing-hematologist-oncologists
#19
REVIEW
Michael A Thompson, Jenny Ahlstrom, Don S Dizon, Yash Gad, Greg Matthews, Howard J Luks, Andrew Schorr
Social media utilizes specific media platforms to allow increased interactivity between participants. These platforms serve diverse groups and purposes including participation from patients, family caregivers, research scientists, physicians, and pharmaceutical companies. Utilization of these information outlets has increased with integration at conferences and between conferences with the use of hashtags and "chats". In the realm of the "e-Patient" it is key to not underestimate your audience...
October 2017: Seminars in Hematology
https://www.readbyqxmd.com/read/29153076/editorial-overview-emerging-importance-of-social-media-for-real-time-communication-in-the-modern-medical-era
#20
EDITORIAL
Naveen Pemmaraju
No abstract text is available yet for this article.
October 2017: Seminars in Hematology
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