The Orange County Convention Center, site of the 2015 ASH Annual Meeting. Photo courtesy of ASH.

The 57th ASH Annual Meeting took place December 5-8, 2015, in Orlando, Florida.

Drug granted orphan designation for SCD

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Sickled and normal
red blood cells
Image by Graham Beards

The US Food and Drug Administration (FDA) has granted orphan drug designation for the small molecule GBT440 to treat patients with sickle cell disease (SCD). GBT440 is being developed as a potentially disease-modifying therapy for SCD. The drug works by increasing hemoglobin’s affinity... [Read Article]

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A new standard of care for rel/ref MM?

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Philippe Moreau, MD
Photo courtesy of ASH

Adding the oral proteasome inhibitor ixazomib to treatment with lenalidomide and dexamethasone can prolong progression-free survival (PFS) in patients with relapsed and/or refractory multiple myeloma (MM), according to interim results of the phase 3 TOURMALINE-MM1 trial. It is not yet clear if the 3-drug combination can prolong... [Read Article]

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High-risk B-ALL subgroup has ‘outstanding outcomes’

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Elizabeth Raetz, MD
Photo courtesy of ASH

A subgroup of young patients with high-risk B-cell acute lymphoblastic leukemia (B-ALL) can have “outstanding outcomes” with contemporary therapy, according to researchers. Results of a large study suggested that patients ages 1 to 30 who have high-risk B-ALL according to National Cancer Institute (NCI) classification can have... [Read Article]

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EZH2 inhibitor can produce durable responses

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Micrograph showing DLBCL

Updated results of a phase 1 study suggest the EZH2 inhibitor tazemetostat can produce durable responses in patients with advanced non-Hodgkin lymphoma (NHL). The drug has demonstrated activity against diffuse large B-cell lymphoma (DLBCL), follicular lymphoma (FL), and marginal zone lymphoma (MZL). The overall response rate among NHL patients in this study... [Read Article]

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BM fibrosis grade may impact OS in PMF

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Micrograph showing PMF

Having a higher grade of bone marrow (BM) fibrosis may confer inferior overall survival (OS) in patients with primary myelofibrosis (PMF), according to a retrospective study. Investigators found that having a fibrosis grade of 2 or higher at diagnosis was associated with “unique clinical and molecular variables” that suggested a more aggressive... [Read Article]

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Frailty in HSCT population not dependent on age

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Woman exercising in a park
Photo by Peter Griffin

Frailty after hematopoietic stem cell transplant (HSCT), while associated with higher mortality, is not necessarily a function of age, according to investigators studying the impact of frailty on transplant outcomes. Instead, other factors, such as increasing time from transplant, employment status,... [Read Article]

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Combo deepens responses and improves PFS in MM

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Jesús San-Miguel, MD, PhD
Photo courtesy of the
University of Navarra

The addition of panobinostat to bortezomib-dexamethasone therapy in relapsed or refractory multiple myeloma (MM) patients can double the rate of deep responses and prolong progression-free survival (PFS), according to an updated analysis of data from the PANORMA 1 trial. Panobinostat, a pan-deacetylase... [Read Article]

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Statin may reduce vaso-occlusive pain in SCD

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Prescription medications
Photo courtesy of the CDC

In a small study, the cholesterol-lowering medication simvastatin reduced the frequency of vaso-occlusive pain in adults and children with sickle cell disease (SCD). Overall, there was a 46% decrease in the frequency of vaso-occlusive pain after 3 months of treatment with simvastatin. There was a slight overall... [Read Article]

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Potential new alternative in CML when TKI therapy fails

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Exhibit hall at the 2015
ASH Annual Meeting
Photo courtesy of ASH

ABL001, an allosteric inhibitor of BCR-ABL1, has shown early evidence of single-agent activity in a multicenter, first-in-human, first-in-class trial of heavily treated patients with chronic myeloid leukemia (CML) that is resistant to or intolerant of prior tyrosine kinase inhibitors (TKIs), even at the... [Read Article]

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Osteoarticular pain affects CML patients stopping TKI

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ASH Annual Meeting attendees
Photo courtesy of ASH

Cases of musculoskeletal pain have been reported after patients stop taking tyrosine kinase inhibitors (TKIs) for chronic myeloid leukemia (CML). TKI discontinuation trials—notably, the STOP imatinib (STIM) trials and EURO-SKI trial—have been conducted to assess the feasibility of maintaining molecular remission once patients discontinue... [Read Article]

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CAR T cells persist for 3 years in young ALL patients

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CTL019 preparation
Photo courtesy of Penn Medicine

CTL019, a CD19 chimeric antigen receptor (CAR) T-cell therapy, can persist for 3 years or longer in children and young adults with relapsed/refractory acute lymphoblastic leukemia (ALL), according to the latest results of a pilot study. This suggests CTL019 can offer long-term disease control without subsequent therapy,... [Read Article]

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Graft source and timing of HSCT affect survival in SCD

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Bone marrow harvest
Photo by Chad McNeeley

In a large, registry-based study, transplants from human leukocyte antigen (HLA)-identical sibling donors proved successful in more than 90% of children and adults with severe sickle cell disease (SCD). However, younger patients and those who received bone marrow (BM) or cord blood (CB) transplants fared the best.... [Read Article]

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Venetoclax produces deep responses in ultra-high-risk CLL

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Stephan Stilgenbauer, MD
Photo courtesy of ASH

The pivotal phase 2 study of venetoclax monotherapy in patients with relapsed/refractory 17p-deleted chronic lymphocytic leukemia (CLL) has achieved unprecedented deep responses, according to investigators. More than 10% of patients had a complete response (CR), complete response with incomplete blood count recovery (CRi), or near partial response... [Read Article]

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SAA patients benefit from upfront eltrombopag combo

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Danielle Townsley, MD
Photo courtesy of ASH

Investigators are pursuing an upfront approval for eltrombopag in combination with immunosuppressive therapy for the treatment of severe aplastic anemia (SAA). Based on eltrombopag’s single-agent activity in refractory SAA, they hypothesized that its addition to standard immunosuppressive therapy of horse antithymocyte globulin (hATG) and cyclosporine (CsA) in... [Read Article]

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Five-year data suggest ruxolitinib improves survival in MF

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Attendees at the 2015
ASH Annual Meeting
Photo courtesy of ASH

Five-year results from the COMFORT-II trial appear to confirm that treatment with ruxolitinib can improve spleen size and survival in patients with myelofibrosis (MF). “These results pave the way to use ruxolitinib earlier in the course of the disease,” said lead study... [Read Article]

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