Approaches to Supportive Care for Blood Disorders Improve Outcomes, Expand Access to Treatment

Dec 05, 2015, 10:30 ET from American Society of Hematology

ORLANDO, Fla., Dec. 5, 2015 /PRNewswire-USNewswire/ -- New research to be presented today at the 57th American Society of Hematology (ASH) Annual Meeting and Exposition provides new answers to common clinical questions and identifies strategies for improving supportive care for patients with blood disorders. 

Supportive care refers to strategies aimed at preventing or treating symptoms of disease or therapy side effects. For patients with blood disorders, supportive care may refer to receiving blood transfusions to manage severe anemia, taking the correct anticoagulant dose that won't result in excessive bleeding or clotting, or ingesting a daily pill to avoid cancer relapse. In all of these scenarios, clinicians must evaluate available evidence to determine the best strategies for their patients. Research to be presented today builds on this evidence in a range of supportive care topics, from blood banking and electronic drug monitoring to managing sometimes fatal blood clotting and bleeding that may be associated with chemotherapy. 

One study examines the common problem of children in remission from acute lymphocytic leukemia (ALL) not adhering to their maintenance drug regimens, thus putting them at risk of relapse. Another describes the first clinical trial comparing blood stored for a prolonged period of time with newer blood. The outcomes of this study (observed in children with severe anemia in Kampala, Uganda) may influence global health policy decisions regarding the acceptable duration of blood storage worldwide, leading to more accessible transfusions for patients who often do not have timely access to blood.

Two additional studies to be presented examine the common problem of cancer-associated thrombosis. One seeks to validate dosing guidelines for avoiding chemotherapy-induced thrombocytopenia, a common autoimmune response to therapy. An additional study compares self-injection to a pill for the long-term treatment of cancer-associated thrombosis. Finally, a late-breaking study demonstrates the best outcomes in 30 years for patients with severe aplastic anemia, achieved by adding orally administered eltrombopag to the standard treatment regimen.

"Research presented today provides important and even surprising insights into the best treatment strategies for patients living with blood diseases. These data will have an immediate clinical impact on patients in a variety of settings worldwide," said Mark Crowther, MD, Professor and Chair in the Department of Pathology and Molecular Medicine of McMaster University in Hamilton, Ontario, Canada. "As the research community better understands the impact and delivery of care, we can help patients with blood disease experience better treatment outcomes and live fuller lives."

This press conference will take place on Saturday, December 5, 2015, at 10:30 a.m. in room W208AB of the Orange County Convention Center.    

Researchers Identify Children Most at Risk of Over-Reporting Adherence to At-Home Chemotherapy Regimen
6-Mercaptopurine (6MP) Intake during Maintenance for Childhood Acute Lymphoblastic Leukemia (ALL) - a Comparison of Self-Report and Electronic Monitoring: A Report from the Children's Oncology Group (COG) Study AALL03N1 [559]

  • 6-Mercaptopurine (6MP) is an oral chemotherapy drug often used to treat acute lymphocytic leukemia (ALL), the most common blood cancer in children. While taking 6MP daily during maintenance therapy as prescribed by a doctor is critical to sustaining durable remissions, not all children take their medicine as directed.
  • Asking patients to self-report their intake of 6MP is convenient, inexpensive, and helps doctors understand if doses are being missed and interventions are needed. However, previous studies not related to oncology show that patients often report that they took their medicine even if they missed doses.
  • This study aimed to compare self-reported 6MP intake with electronic monitoring to identify predictors of over-reporting of 6MP intake.
  • Participants included 416 children with ALL in their first remission who were prescribed oral 6MP. The patients' ages ranged from 2 to 20 years old.
  • Treatment intake for each patient was measured electronically using a system that recorded each date and time the 6MP bottle was opened during a span of four months. At the end of each of the four months, the child or parent reported the number of days that 6MP had been taken during the preceding month.
  • Patients were then categorized as perfect reporters if the self-report matched the electronic report, over-reporters if the self-report indicated more doses than the electronic report by five or more days per month for more than half of months of monitoring, or "others."
  • Only 12 percent of patients were identified as perfect reporters; 23.6 percent were over-reporters, and 64.4 percent were considered "others."
  • Upon further analysis, researchers found that for each increasing year of age of the child, the risk of over-reporting increased by seven percent. Race was also a factor, as Hispanic children/parents were 2.4 times more likely to over-report, Asians were 3.1 times more likely to over-report, and African Americans were 5.3 times more likely to over-report when compared with non-Hispanic white children/parents. Children whose father did not attend college were 2.1 times more likely to over-report.
  • Finally, children who did not adhere to their 6MP regimen (i.e., those whose adherence rate was less than 95% of prescribed doses) were 8.6 times more likely to overstate their monthly doses, when compared with children who adhered to prescribed 6MP. While 78.6 percent of over-reporters were non-adherent, only one of 50 (2%) of the perfect reporters were non-adherent.
  • Researchers conclude that approximately 25 percent of children/parents over-report 6MP intake; and over-reporting is more likely in patients who are non-adherent to 6MP, older, of a minority race, and from households with lower paternal education.
  • These findings suggest that subjective reporting of 6MP ingestion during maintenance therapy for childhood ALL should be used with caution.

Wendy Landier, PhD, University of Alabama at Birmingham, Ala., will present this study during an oral presentation on Saturday, December 5, at 12:45 p.m. in room W230, level 2 of the Orange County Convention Center.

Study Finds No Difference Between Older and Newer Stored Blood
Tissue Oxygenation by Transfusion in Severe Anemia with Lactic Acidosis (TOTAL): A Prospective, Randomized, Non-Inferiority Trial of Blood Storage Duration [769]

  • Transfusion of Red Blood Cells (RBCs), the process of infusing a patient with donated blood, aims to increase oxygen delivery to tissues. 
  • During refrigerated blood storage, RBCs undergo changes that collectively might diminish the blood's ability to transfer oxygen to tissues. Current regulations allow RBCs to be stored in approved solutions for up to five or six weeks. However, concerns regarding changes in RBCs during storage have questioned whether the allowable storage limit should be shortened.
  • A reduction in the shelf-life of RBCs would reduce blood availability especially in developing nations where blood supplies are already limited.
  • Study authors conducted the first large randomized, controlled clinical trial comparing the oxygen delivery of short-storage versus longer-storage RBCs as measured by transfused patients' blood lactate levels. When tissue oxygen levels are critically low, lactate levels rise, and when tissues are successfully re-oxygenated, lactate levels may fall again. 
  • The study examined 290 patients between the ages of six months and five years who visited a university hospital urgent care facility in Kampala, Uganda, for treatment of severe anemia marked by elevated blood lactate levels.
  • Patients were randomly assigned to receive either RBCs stored for one to 10 days, or RBCs stored for 25 to 35 days.
  • Patients were monitored from start of transfusion to 24 hours following transfusion.
  • The number of patients achieving the desired blood lactate level of less than or equal to 3mM was not statistically different between the two groups. Of those who received shorter-storage blood, 58 percent reached this desired outcome compared to 61 percent of those who received longer-storage blood.
  • Brain oxygen levels increased in recipients of shorter-storage and longer-storage RBCs to the same degree. 
  • Researchers conclude that longer-storage RBCs are not inferior to shorter-storage RBCs. These results may have significance for global health policy decisions regarding the acceptable duration of RBC storage.

Christine M. Cserti-Gazdewich, MD, University Health Network, Toronto, Ontario, Canada will present this study during an ASH press conference on Saturday, December 5, at 10:30 a.m. Walter Dzik, MD, Massachusetts General Hospital, Boston, will present this study during an oral presentation on Monday, December 7, at 4:30 p.m. in room W308, level 3 of the Orange County Convention Center.

Research Provides Guidance for Anticoagulation Management in the Setting of Thrombocytopenia in Cancer Patients
Enoxaparin Dose Reduction for Thrombocytopenia in Patients with Cancer: A Quality Assessment Study [429]

  • Cancer patients commonly develop thrombosis and are often prescribed therapeutic anticoagulants while on chemotherapy.
  • Thrombocytopenia, a condition characterized by a deficiency of platelets in the blood, is a common adverse event associated with chemotherapy. Patients with chemotherapy-induced thrombocytopenia (CIT) have an increased risk of bleeding.
  • Managing anticoagulation in cancer patients who have CIT is challenging because clinicians must find a balance between the risks of both bleeding and recurrent thrombosis.
  • The current standard of care for preventing blood clots in a cancer setting includes treatment with low-molecular-weight heparin (LMWH), a type of anticoagulant. However, there is limited evidence to support proper dosing to treat these episodes in the setting of CIT. In 2010, Memorial Sloan Kettering Cancer Center (MSK) implemented guidelines for LMWH dose modifications in the setting of thrombocytopenia.
  • In order to evaluate the safety and efficacy of these guidelines, researchers conducted a retrospective analysis to evaluate the outcomes of cancer patients who were thrombocytopenic for at least seven days and received a therapeutic dose of LMWH from 2011 to 2013.
  • Investigators identified 102 patients with a collective 143 episodes of thrombocytopenia.  LMWH dose was modified in 136 of the 144 episodes (95%), which showed adherence to the institutional guidelines.
  • LMWH doses were reduced in 20 episodes, withheld in 89 episodes, and managed with a combination of reduction or withheld in 27 episodes. In general, the more severe thrombocytopenic episodes were managed with holding LMWH as opposed to dose reduction.
  • None of the patients experienced recurrent thrombosis or major bleeds when the anticoagulant management guidelines were followed.
  • The data support the safety and efficacy of following the MSK guidelines for therapeutic LMWH dose modification, balancing the dual risks of recurrent thrombosis and potential bleeding during periods of CIT in cancer patients. More research is needed to see if a similar strategy would be appropriate for other oral anticoagulants.

Gerald Soff, MD, Memorial Sloan Kettering Cancer Center, New York, will present this study during a press conference on Saturday, December 5, at 10:30 a.m.  Yimei Miao, Memorial Sloan Kettering Cancer Center, New York, will present this study during an oral presentation on Monday, December 7, at 7:30 a.m. in room W311ABCD, level 3 of the Orange County Convention Center.

Study Suggests Warfarin is Acceptable for Long-Term Treatment of Cancer-Associated Thrombosis
Switching to Warfarin after 6-Month Completion of Anticoagulant Treatment for Cancer-Associated Thrombosis [430]

  • The standard treatment for cancer patients who develop blood clots is three to six months of anticoagulant therapy with low-molecular-weight heparin (LMWH). However, there are little data to support the best ongoing anti-clotting therapy beyond six months.  
  • This study evaluated the records of 1,502 patients with cancer-associated thrombosis. Investigators selected patients who had completed treatment with LMWH for six months and divided them into two groups – one group of 763 patients continued to receive LMWH and the other group of 739 patients was switched to warfarin, another type of anticoagulant. Because LMWH requires self-injections, warfarin is often thought to be preferred because it can be taken in pill form.
  • The cumulative incidence of major bleeding was 2.6 percent in the LMWH group and 2.7 percent in the warfarin group.
  • The cumulative incidence of total bleeding was 6.7 percent in the LMWH group and seven percent in the warfarin group.
  • The study found that switching to warfarin is not associated with an increase in recurrent VTE, major bleeding, or total bleeding when compared to continuing LMWH. Researchers concluded that warfarin is an acceptable alternative for LMWH for patients with cancer-associated thrombosis.

Chatree Chai-Adisaksopha, MD, McMaster University, Hamilton, Ontario, Canada, will present this study during an oral presentation on Monday, December 7, at 7:45 a.m. in room W311ABCD, level 3 of the Orange County Convention Center.

Adding Eltrombopag to Standard Severe Aplastic Anemia Treatment Improves Responses for First Time in 30 Years
Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia Accelerates Count Recovery and Increases Response Rates [LBA-2]

  • Severe aplastic anemia (SAA) occurs when the bone marrow does not produce enough new blood cells. Affected patients have anemia, thrombocytopenia, and neutropenia; they feel fatigued and are at risk of serious infections and bleeding.
  • The standard of care for SAA is hematopoietic stem cell transplantation or immunosuppressive treatment (IST) with horse antithymocyte globulin and cyclosporine. Despite efforts to improve outcomes for patients with SAA, response rates to IST have remained stable for the last thirty years (60-65% of patients achieve overall hematologic response and approximately 10% achieve complete response, in which the blood counts normalize).
  • Eltrombopag (EPAG) is an oral medication that on its own has been shown to improve blood counts in about 40 percent of patients.
  • In this Phase II trial, NIH researchers examined if adding EPAG to standard IST could further improve this regimen.
  • Eighty-eight patients with previously untreated SAA were enrolled from July 2012 to October 2015. Patients were divided into three cohorts of approximately 30 patients. All received standard IST with daily 150 mg doses of EPAG, administered for different intervals. The primary endpoint was complete remission (CR) at six months.
  • Across all cohorts, the CR rate at six months was 33, 26, and 54 percent, for groups one, two, and three, respectively. For all patients, the rate of CR was higher compared to past studies with standard IST.
  • Overall hematologic response rate was 80, 87, and 92 percent, respectively, for groups one, two, and three.
  • All patients experienced robust increased production of blood cells. The median time to transfusion-independence for platelets was 32 days and for red cells 42 days. For patients with severely low white blood cell counts, the median time to reconstitution to adequate white blood cell count was 47 days.
  • The treatment was well tolerated by the majority of patients. Two patients discontinued EPAG because of severe skin reactions and six patients withdrew before six months because their disease progressed or resisted treatment.
  • The addition of EPAG to standard treatment notably increased the rate of complete hematologic response, suggesting that EPAG combined with IST may accelerate the rate and quality of hematopoietic recovery.
  • The rapid blood count improvement supports early use of EPAG and IST for patients with newly diagnosed SAA.

Danielle M. Townsley, MD, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Md., will present this study during the Late-Breaking Abstracts Session on Tuesday, December 8, at 7:30 a.m. in Hall D, level 2 of the Orange County Convention Center.

American Society of Hematology 57th Annual Meeting
The study authors and press program moderator will be available for interviews after the press conference or by telephone. Additional press briefings will take place throughout the meeting on gene therapy, precision medicine, novel therapies for blood cancers, and sickle cell disease. For the complete annual meeting program and abstracts, visit Follow @ASH_hematology and #ASH15 on Twitter and like ASH on Facebook for the most up-to-date information about the 2015 ASH Annual Meeting.

The American Society of Hematology (ASH) ( is the world's largest professional society of hematologists dedicated to furthering the understanding, diagnosis, treatment, and prevention of disorders affecting the blood. For more than 50 years, the Society has led the development of hematology as a discipline by promoting research, patient care, education, training, and advocacy in hematology. The official journal of ASH is Blood (, the most cited peer-reviewed publication in the field, which is available weekly in print and online and has been serving the hematology community for 70 years.


SOURCE American Society of Hematology