CLINICAL NEWS
World Federation of Hemophilia Creates Universal Case Report Form for Hemophilia Patients
The World Federation of Hemophilia ( WFH ) created the Universal Case Report Form ( U-CRF ) to collect and standardize data on relevant diagnosis , therapies , and outcomes for patients with hemophilia .
Previous WFH forms only provided an overview of the number of patients and access to treatment , and did not include information on how treatments were performed or on patient outcomes .
The form is meant to help hemophilia treatment centers ( HTCs ) provide more comprehensive data on patients , increase access to therapy , improve the quality of care , and enable the comparison of outcomes among different patient groups .
U-CRF is composed of three parts to account for differences in clinical practices at HTCs around the world :
• Part 1 : the Registration Form , which provides information on the location , contact and responsible personnel , and number of registered patients in each HTC
• Part 2 : the Minimal Data Set includes basic information such as patient demographics , diagnosis data at registration , annual followup with details of bleeding , chlorofluorocarbons replacement , and inhibitor status
• Part 3 : includes full details of the relevant parameters in patient outcome
The report is available at wfh . org / en / u-crf .
Source : WFH press release , September 2 , 2016 .
FDA Approves Blinatumomab for Pediatric Acute Lymphocytic Leukemia
The U . S . Food and Drug Administration ( FDA ) approved blinatumomab for pediatric and adolescent patients with Philadelphia chromosome-negative relapsed / refractory B-cell precursor acute lymphocytic leukemia ( ALL ).
The approval was based on the results of a single-arm , openlabel , multicenter , dose-finding phase I / II study , in which patients receiving blinatumomab achieved complete remission ( CR ) within the first two cycles of treatment ( the study ’ s primary endpoint ).
A total of 93 pediatric and adolescent patients (< 18 years ) who had refractory disease relapsed at least twice , or relapsed after an allogeneic hematopoietic cell transplantation were included in the study . During phase I , the researchers proposed a stepwise dosing of 5 / 15 μg / m ² per day .
During phase I , the CR rate was approximately 32 percent among 41 patients who were enrolled . Of those patients , 77 percent reached a minimum residual disease ( MRD ) -negative state . The median relapse-free survival ( RFS ) was 8.3 months , and the median overall survival ( OS ) was 5.7 months .
Among 39 patients enrolled in the phase II study , the CR rate was 31 percent ( n = 12 ). Of those patients , 42 percent were MRDnegative . The median RFS was 5.6 months , and the median OS was 4.3 months .
The most common treatmentrelated grade ≥3 adverse events included anemia , thrombocytopenia , febrile neutropenia , hypokalemia , and neutropenia .
Patients are undergoing continued assessment for long-term efficacy outcomes .
Source : Amgen news release , September 1 , 2016 .
FDA Grants Pracinostat Breakthrough Designation for Older Patients With AML
The U . S . Food and Drug Administration ( FDA ) granted breakthrough therapy designation for pracinostat in combination with azacitidine for patients with newly diagnosed acute myeloid leukemia ( AML ) who are ≥75 years old or ineligible for intensive chemotherapy .
The FDA ’ s decision was based on the results of a phase II trial that reported a complete response rate ( primary endpoint ) of 42 percent and a median overall survival ( secondary endpoint ) of 19.1 months among treatment-naïve older AML patients treated with pracinostat and azacitidine .
A total of 50 patients were enrolled at 15 study locations between December 2013 and December 2015 . The median patient age was 75 years ( range = 66-84 years ) and , at baseline , the median bone marrow blast count was 40 percent ( range = 20-89 %).
Patients received 28-day cycles of the following regimen until disease progression , unacceptable toxicity , or no response :
• 60 mg of oral pracinostat administered on three alternating days each week for three weeks
• 75 mg / m 2 of azacitidine administered intravenously or subcutaneous on days one through seven or one through five and eight through nine
The most common treatment-related grade ≥3 adverse events ( AEs ) included febrile neutropenia ( 30 %), thrombocytopenia ( 22 %), neutropenia ( 10 %), cellulitis ( 10 %), anemia ( 8 %), fatigue ( 8 %), sepsis ( 6 %), and pancytopenia ( 6 %). Seven patients experienced AEs that led to treatment discontinuation , including peripheral motor neuropathy ( n = 1 ), parainfluenza ( n = 1 ), atrial fibrillation / prolonged QTc ( n = 1 ), subdural hematoma after a fall ( n = 1 ), and sepsis ( n = 3 ).
Source : FDA news release , August 1 , 2016 .
NHLBI Releases Strategy for Heart , Lung , Blood , and Sleep Research
The National Heart , Lung , and Blood Institute ( NHLBI ) announced its Strategic Vision ( nhlbi . nih . gov / about / documents / strategic-vision ) to chart the course of heart , lung , blood , and sleep research over the next 10 years .
After collaborating with various stakeholders , including the American Society of Hematology , and engaging more than 4,500 respondents from all 50 U . S . states , as well as 42 countries , the NHLBI outlined the following four main goals :
• understand human biology
• reduce human disease
• advance translational research
• develop workforce and resources
With innovations in areas such as computational biology , data science , bioengineering , and other technologies , NHLBI hopes that the initiative will provide more information on :
• the complex interplay of environmental , behavioral , and molecular factors that promote health
• the earliest point of disease development
• the ability to repair damaged hearts with stem cell and tissue engineering techniques
• a new generation of novel therapeutics
• the ability to develop cures through gene editing and stem cell technology for sickle cell disease and other conditions ●
Source : NHLBI press release , August 11 , 2016 .
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