ASH 2015

 
 
 

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

HT Staff Read Article
Published: 01/05/16

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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 for oxygen. Since oxygenated sickle hemoglobin does not polymerize, it is believed that GBT440 blocks polymerization and the resultant sickling of red blood cells. [Read Article]

A new standard of care for rel/ref MM?

Jen Smith Read Article
Published: 01/03/16

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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 overall survival when compared to treatment with lenalidomide and dexamethasone.  [Read Article]

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

Jen Smith Read Article
Published: 01/02/16

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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 high rates of event-free survival (EFS) and overall survival (OS) if they have favorable cytogenetic . . . [Read Article]

EZH2 inhibitor can produce durable responses

Jen Smith Read Article
Published: 12/31/15

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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 was 56%, and 1 patient has maintained a response for more than 21 months. [Read Article]

BM fibrosis grade may impact OS in PMF

Jen Smith Read Article
Published: 12/31/15

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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 disease phenotype. And the median OS was significantly shorter in patients with higher grades of fibrosis. [Read Article]

Frailty in HSCT population not dependent on age

Erilyn Riley Read Article
Published: 12/30/15

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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, medical leave or disability, and limitations of social activities, were significantly associated with higher odds of frailty. [Read Article]

Combo deepens responses and improves PFS in MM

Mark Fuerst Read Article
Published: 12/30/15

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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 inhibitor, was the first agent of its class to produce a statistically significant and clinically meaningful increase in the median PFS . . . [Read Article]

Statin may reduce vaso-occlusive pain in SCD

Jen Smith Read Article
Published: 12/23/15

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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 decrease in the intensity of pain as well, but this was not statistically significant. [Read Article]

Potential new alternative in CML when TKI therapy fails

Erilyn Riley Read Article
Published: 12/21/15

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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 lowest dose evaluated. ABL001 and classical TKIs exhibit complementary mutation profiles, with ABL001 showing activity against TKI resistance mutations. [Read Article]

Osteoarticular pain affects CML patients stopping TKI

Erilyn Riley Read Article
Published: 12/18/15

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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 a TKI. However, none of the studies collected low-grade events before or after patients discontinued TKI therapy. [Read Article]

Venetoclax produces deep responses in ultra-high-risk CLL

Erilyn Riley Read Article
Published: 12/17/15

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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 (nPR), as confirmed by an independent review committee (IRC). [Read Article]

CAR T cells persist for 3 years in young ALL patients

Mark Fuerst Read Article
Published: 12/17/15

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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, such as stem cell transplant, said study author Stephan Grupp, MD, PhD, of the University of Pennsylvania in Philadelphia. [Read Article]

Graft source and timing of HSCT affect survival in SCD

Jen Smith Read Article
Published: 12/17/15

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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. Patient age and stem cell source were both independently associated with event-free and overall survival. [Read Article]

SAA patients benefit from upfront eltrombopag combo

Erilyn Riley Read Article
Published: 12/16/15

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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 the first-line setting could improve patient outcome. And, in a phase 2 trial, it did. [Read Article]

Five-year data suggest ruxolitinib improves survival in MF

Mark Fuerst Read Article
Published: 12/16/15

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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 author Claire Harrison, MD, a consultant hematologist at Guy’s and St. Thomas’ NHS Foundation Trust in London, UK. Dr Harrison presented the results at the 2015 ASH Annual Meeting. [Read Article]

Regimen with intensified PEG-ASP feasible in young adults with ALL

Erilyn Riley Read Article
Published: 12/16/15

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Crowd at the 2015 ASH
Annual Meeting
Photo courtesy of ASH

Results of a DFCI ALL Consortium trial have shown that adults with acute lymphoblastic leukemia (ALL) can be successfully and safely treated with a pediatric regimen using intensified pegylated asparaginase (PEG-ASP). Investigators recently reported that young adults treated with native E coli asparaginase as part of their regimen had improved 4-year disease-free survival and overall survival (OS) rates.  [Read Article]

Surprising finding in upfront use of idelalisib monotherapy

Erilyn Riley Read Article
Published: 12/15/15

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Attendees at ASH 2015
Photo courtesy of ASH

Investigators have observed early fulminant hepatotoxicity in a subset of primarily younger chronic lymphocytic leukemia (CLL) patients treated with idelalisib monotherapy in the frontline setting. In a phase 2 study of idelalisib plus ofatumumab, 52% of the 24 patients enrolled experienced grade 3 or higher hepatotoxicity shortly after idelalisib was started. [Read Article]

CAR T-cell therapy dubbed ‘promising’ for MM

Jen Smith Read Article
Published: 12/15/15

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James Kochenderfer, MD
Photo courtesy of ASH

Chimeric antigen receptor (CAR) T cells can have “powerful activity” in patients with multiple myeloma (MM), according to a speaker at the 2015 ASH Annual Meeting. The CAR T cells in question are directed against the B-cell maturation antigen (BCMA), a protein expressed by normal and malignant plasma cells. In a phase 1 study of patients with previously treated MM, CAR-BCMA T cells eliminated plasma cells without causing direct damage to essential organs. [Read Article]

HU noninferior to transfusion for stroke prevention in SCD

Jen Smith Read Article
Published: 12/14/15

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Russel Ware, MD, PhD
Photo courtesy of ASH

Hydroxyurea (HU) is noninferior to chronic blood transfusions for reducing the risk of stroke in children with sickle cell disease (SCD), results of the TWiTCH trial suggest. The trial showed that daily doses of HU lower the transcranial Doppler (TCD) blood velocity in children with SCD to a similar degree as blood transfusions, thereby decreasing the risk of stroke. Because of these findings, the trial was terminated early, in November of last year. [Read Article]

Combination offers ‘important new option’ for CLL, team says

Erilyn Riley Read Article
Published: 12/13/15

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Attendees meet in Orlando
Photo courtesy of ASH

Idelalisib, the first-in-class PI3Kδ inhibitor, combined with bendamustine and rituximab (BR) for relapsed/refractory chronic lymphocytic leukemia (CLL) offers “an important new option over the standard of care,” according to Andrew Zelenetz, MD, a member of the international research team that conducted the phase 3 study of this combination. Patients who received idelalisib plus BR experienced a much longer progression-free survival (PFS) than . . . [Read Article]

Group recommends adding rituximab to ALL therapy

Erilyn Riley Read Article
Published: 12/12/15

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Sebastien Maury, MD
Photo courtesy of ASH

Investigators from the Group for Research on Adult Lymphoblastic Leukemia (GRAALL) recommend integrating rituximab into the treatment of adult patients with acute lymphoblastic leukemia (ALL) based on results of the GRAALL-R 2005 study. Patients who received rituximab as part of their therapy had a median event-free survival (EFS) at 2 years of 65% compared to 52% of patients who did not receive rituximab. [Read Article]

RATIFY trial results available after almost 10 years in the making

Erilyn Riley Read Article
Published: 12/11/15

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Richard Stone, MD
Photo courtesy of ASH

After almost 10 years, during which time no new drug for acute myeloid leukemia (AML) has been approved, results of the RATIFY trial are available. In this trial, investigators evaluated midostaurin in combination with chemotherapy for younger patients with FLT3-mutated AML. Patients who received midostaurin in their regimen instead of placebo experienced significantly longer overall survival (OS) and event-free survival (EFS). [Read Article]

Triplet disappoints in follicular lymphoma trial

Jen Smith Read Article
Published: 12/11/15

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2015 ASH Annual Meeting
Photo courtesy of ASH

A 3-drug regimen is likely not worth pursuing as a first-line treatment option for follicular lymphoma (FL), according to a presentation at the 2015 ASH Annual Meeting. In a phase 1 study, combination ibrutinib, rituximab, and lenalidomide did not provide any response benefit over that previously observed with rituximab and lenalidomide. But the triplet increased toxicity—particularly the incidence of rash—and necessitated dose modifications.  [Read Article]

Rivaroxaban performs as expected in clinical practice

HT Staff Read Article
Published: 12/11/15

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Rivaroxaban

“Real-world” data appear to confirm phase 3 results with rivaroxaban in patients who have deep vein thrombosis (DVT), with or without concomitant pulmonary embolism (PE). Results of the phase 4 XALIA study suggest that, in clinical practice, the rates of major bleeding and recurrent venous thromboembolism (VTE) in patients taking rivaroxaban are generally consistent with results of the phase 3 EINSTEIN-DVT study. [Read Article]

mAb could provide targeted approach to HLH treatment

Jen Smith Read Article
Published: 12/10/15

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Attendees at the 2015
ASH Annual Meeting
Photo courtesy of ASH

A monoclonal antibody (mAb) targeting interferon-gamma (IFNγ) has shown promise for treating hemophagocytic lymphohistiocytosis (HLH), according to a late-breaking abstract presented at the 2015 ASH Annual Meeting. Results of an ongoing phase 2 study suggest the mAb, NI-0501, may be a feasible treatment option for patients with HLH who have demonstrated an unsatisfactory response to, or cannot tolerate, conventional therapy. [Read Article]

Source of FVIII replacement matters, study shows

Jen Smith Read Article
Published: 12/10/15

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Flora Peyvandi, MD, PhD
Photo courtesy of ASH

The source of factor VIII (FVIII) replacement therapy affects the risk of inhibitor development in previously untreated patients with severe hemophilia A, according to a prospective, randomized trial. Results of the SIPPET study indicate that receiving recombinant FVIII is associated with a nearly 2-fold higher risk of developing inhibitory alloantibodies than receiving plasma-derived FVIII.  [Read Article]

2nd-gen BTK inhibitor may be safer, team says

HT Staff Read Article
Published: 12/10/15

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Micrograph showing CLL

The second-generation BTK inhibitor acalabrutinib (ACP-196) can elicit durable partial responses in patients with chronic lymphocytic leukemia (CLL) while producing minimal side effects, according to researchers. They said data suggest that, compared to the first-generation BTK inhibitor ibrutinib, acalabrutinib more selectively blocks the BTK pathway. And it does so without disrupting other molecular pathways that are important . . . [Read Article]

CAR exhibits activity in resistant B-cell malignancies

Jen Smith Read Article
Published: 12/09/15

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James N. Kochenderfer, MD
Photo courtesy of ASH

Allogeneic chimeric antigen receptor (CAR) T cells can have “significant” activity against resistant B-cell malignancies, even when given without prior chemotherapy, according to a presentation at the 2015 ASH Annual Meeting. Nine of 20 patients responded to treatment with the CAR T cells, despite having failed prior allogeneic transplant. The best responses were observed in patients with acute lymphoblastic leukemia (ALL) and chronic lymphocytic leukemia (CLL). [Read Article]

RBC storage duration doesn’t affect outcomes in kids

HT Staff Read Article
Published: 12/08/15

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Blood for transfusion
Photo by Elise Amendola

The storage duration of red blood cells (RBCs) doesn’t affect transfusion outcomes in children with lactic acidosis due to severe anemia, according to a new study. Investigators found no significant differences in lactate levels, 30-day recovery, survival, or adverse events between children who received RBCs stored for 25 to 35 days and children who received RBCs stored for 1 to 10 days.  [Read Article]

Ibrutinib ‘treatment of choice’ in rel/ref MCL

Jen Smith Read Article
Published: 12/08/15

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Crowd at 2015 ASH
Annual Meeting
Photo courtesy of ASH

The BTK inhibitor ibrutinib should be considered the treatment of choice for patients with relapsed or refractory mantle cell lymphoma (MCL), according to a speaker at the 2015 ASH Annual Meeting. Results of the phase 3 RAY trial showed that ibrutinib can prolong progression-free survival (PFS) when compared to the mTOR inhibitor temsirolimus. There was no significant difference in overall survival (OS) between the treatment arms . . . [Read Article]

ALL patients over-report their 6MP compliance, researchers say

Erilyn Riley Read Article
Published: 12/08/15

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Wendy Landier, PhD, CRNP
Photo courtesy of ASH

A study comparing subjective versus objective reporting of treatment compliance in patients with acute lymphoblastic leukemia (ALL) has shown that about a fourth of patients over-report how compliant they are with taking 6-mercaptopurine (6MP) as part of their maintenance therapy. An earlier analysis of the COG AALL03N1 compliance study showed that adherence rates of less than 95% were associated with a 3.7-fold increased risk of relapse. [Read Article]

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