2010 Call for Abstracts

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The 52nd Annual Meeting of the American Society of Hematology (ASH) will be held Saturday, December 4, through Tuesday, December 7, 2010, in Orlando, Florida. The goal of the ASH annual meeting is to present the best new scientific research in all areas of hematology.

All abstract submissions must be made electronically through ASH’s online abstract submission system. The site opens on June 14, and the abstract submission deadline is Thursday, August 12, 2010, 11:59 p.m. (PDT).

Eligibility

To submit an abstract, the following criteria must be met:

  • At least one of the authors must be an ASH member who has paid the current membership dues.
  • If none of the abstract authors is an ASH member, the abstract must be sponsored by a current ASH member, or it will not be allowed to be submitted. ASH members are urged to use their best judgment in restricting sponsorship to a reasonable number of abstracts, keeping in mind that they are endorsing the authenticity and quality of each abstract that they sponsor.
  • Research and/or studies must fit into one of the ASH 2010 Abstract Review Categories.
  • Data already published by August 12 either online or in print, data presented at a major national or international meeting, and data to be presented at an ASH Friday Satellite Symposium will generally not be eligible for consideration. We encourage the authors to request special consideration if the abstract is a minor extension of published work or if the author has a reason to believe that the data will be published, either online or in print, or presented prior to the ASH annual meeting.

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Responsibilities of the Presenting Author

  • The first author listed for each abstract serves as the presenting author and as the primary contact for all correspondence regarding the abstract, unless otherwise specified under the “Contact Information” section of the online abstract submission system.
  • The presenting author must be one of the co-authors listed on the submitted abstract.
  • The presenting author is responsible for the following:
    • Ensuring that all authors have read the abstract and agreed to be co-authors. Failure to get approval from all authors will result in rejection of the abstract.
    • Notifying all co-authors of any additions, deletions, and changes to the program, as may be communicated by ASH.
    • Obtaining all of the conflict-of-interest disclosure and copyright transfer information from co-authors.
    • Forwarding all correspondence to all co-authors, including ASH policies and guidelines and the Accreditation Council for Continuing Medical Education (ACCME) Standards for Commercial Support for Continuing Medical Education (CME).

ASH Statement on Commercial Interest Presenters

The primary purpose of the ASH Continuing Medical Education Program is to advance the professional development of our learners in order to increase knowledge and improve the diagnosis and treatment of patients. In order to maintain the flow of the most current information to our learners and to serve the public interest, the content of ASH CME may include information planned, presented, or authored by employees of commercial interests. ASH is committed to the integrity of the science presented at its CME activities and employs a rigorous peer-review process to ensure that integrity. The content of presentations by commercial interest employees must focus on basic science and not on the product or on the commercial aspects of the discovery. In addition, the format of the presentation must permit full discussion of the therapeutic benefits and risks of the discovery. ASH maintains control of all content and plans all of its activities in compliance with the ACCME Standards for Commercial Support.

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Authors' Consent and Waiver of Claims

  • Each abstract author agrees and certifies that he or she:
    • has read all of the rules and agrees to be bound by them,
    • is responsible for submission of the abstract in accordance with the rules, and
    • waives any and all claims against ASH and any reviewer arising out of or relating to the abstract submission and review process, including but not limited to peer review and the grading of abstracts.

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General Guidelines

  • The abstract must address scientific questions, detail clinical observations, or contain primary scientific data.
  • Abstracts submitted to ASH are embargoed from the time of submission. This means that the data in the abstract cannot be submitted to other meetings and/or publishers once submitted for the ASH annual meeting. Read the “Embargo Policy” section of the Call for Abstracts for more information.
  • All research and studies reported in submitted abstracts that involve human and animal subjects must comply with the guiding principles for experimental procedures found in the Declaration of Helsinki of the World Medical Association.
  • Data from the long-term follow-up of previously presented clinical trials may be submitted only if significant new information can be shown. In this case, please ask for special consideration.
  • Interim analysis of a prospective randomized clinical trial will be considered only if it is performed as planned in the original protocol and is statistically valid. If your abstract involves interim analysis, explain the details of your study in the text box provided under the submission information section in the online abstract submission system.
  • There is a $60 nonrefundable handling fee for submitting an abstract. Payment must be made by credit card; Visa, MasterCard, and American Express are accepted. Purchase orders and checks will not be accepted. The abstract submission fee does not include registration for the annual meeting; therefore, all authors planning to attend the ASH annual meeting must register for the meeting.
  • No revisions can be made after the abstract submission deadline.
  • Abstracts generally may not be withdrawn once submitted. If you prefer that your abstract be withdrawn if not accepted for any presentation at the ASH annual meeting, you must indicate so during online submission.
  • The annual meeting presentation must reflect the abstract. The presentation title, authorship, and scientific content must be the same as the abstract; however, updates on results may be added.
  • Abstracts should be written in clear and concise English, so that reviewers are able to focus solely on the scientific merits of the submission. We encourage non-English-speaking authors to have their abstracts checked for grammar and spelling prior to submission.
  • It is assumed that the presenting author will have adequate command of English to present and to respond to questions. If that is not the case, the authors should choose a poster as their preference.

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Preferred Presentation Format

During submission, submitters must choose the formats in which they would be willing to present/publish their abstract. These choices must be made at the time of submission and cannot be changed after the review committee has reached its decision.

  • First, authors select whether they prefer oral or poster presentation. Abstracts noted with a preference for poster presentation will NOT be considered for oral presentation, regardless of score.
  • Second, in the event that an abstract is not chosen for oral presentation, it can still be chosen for poster presentation. Authors who state a preference for oral presentation must decide whether they would accept poster presentation. Authors may either present a poster or request that the abstract be immediately withdrawn. (Please note that declining to have your abstract considered for poster presentation does not improve its chances of being accepted as an oral presentation. This option should be selected primarily by authors who would not be willing to present a poster.)
  • Finally, all submitting authors must decide whether - in the event the abstract is NOT selected for presentation - they want their abstract to be published in the online-only issue of Blood (which is part of the permanent Blood archive), or whether the abstract should be withdrawn completely and not published either online or in print.

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Requesting Special Consideration

  • Special consideration may be requested by explaining the special circumstances in the text box provided during the online abstract submission process. We encourage authors to request special consideration if the abstract is a minor extension of previously published work or if the author has a reason to believe that the data will be published, either online or in print, or presented in any form prior to the ASH annual meeting. The information provided will be carefully evaluated by the reviewers. ASH reserves the right to deny special consideration and exclude the abstract from the program.
  • Failure of authors to request special consideration for an abstract, including content that has been published or presented at a major national or international meeting or in a press release published before the ASH annual meeting, will result in exclusion from the program.
  • If special consideration is requested for the presentation of previously published data, ASH reserves the right to exclude the abstract from the program.

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Abstract Review and Selection Process

  • After the submission deadline, all completed and eligible abstracts will be made available to the ASH Abstract Reviewers for blinded review and scoring, and final decisions will be made by the Program Committee in late September 2010.
  • Abstracts will be evaluated and scored solely on their scientific merits.
  • Incomplete abstracts will not be reviewed.
  • The same study must not be submitted as multiple abstracts. Abstracts that are simply different versions of a single study will be rejected.
  • Abstracts will be peer reviewed according to the subject categories. Authors must indicate during online submission the appropriate review category (one only). You will be able to use the list of abstract review categories, which provides detailed descriptions of each category, to assist you in selecting the correct abstract classification. Please read through all of the categories, and select the category most closely associated with your abstract. All category selections will be final. There will be NO re-classification of abstracts after the abstract submission site has been closed.
  • All abstracts submitted will be considered eligible for one presentation: either plenary, oral (simultaneous session), or poster.
    • The Plenary Session features the top six abstracts. These are formal oral presentations, each followed by a brief discussion. An ASH member selected by the Program Committee will give a brief introduction to the abstract.
    • Oral (simultaneous) Sessions are formal oral presentations followed by a brief discussion.
    • Poster Sessions allow the viewing of a poster illustration of the abstract. Authors are expected to post and remove posters at designated times and to be at their posters to answer questions during the time designated for poster presentations. The author’s attendance during poster presentation time will be monitored.

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Acceptance/Rejection Notification

  • Notification regarding acceptance or rejection of abstracts will be sent to the presenting author in early October 2010 by e-mail; consequently, an accurate e-mail address is critical. If your abstract is accepted, the e-mail will specify whether it is accepted as plenary, oral, or poster presentation or whether it has been selected to be published online only. If you have not received an e-mail notification by October 7, 2010, contact ASHabstracts@hematology.org. Rejection notifications will also be sent at that time.
  • Important note: To ensure that you are able to receive e-mail correspondence from ASH, please make sure that your e-mail software can receive mail from the confex.com and hematology.org domains. You should add ash@confex.com and ASHAbstracts@hematology.org to your address book. If after completing your submission you don't receive a confirmation e-mail from the abstract system, you must contact your system administrator and make sure that both the confex.com and hematology.org domains are added to your e-mail address whitelist.
  • The decision of the ASH Program Committee regarding acceptance and presentation of abstracts is final.

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Abstract Withdrawal

  • Once an abstract is accepted, a written request to withdraw must be submitted no later than October 8, 2010, to ASHabstracts@hematology.org if the first author decides to withdraw the abstract for any reason. Abstract withdrawal requests received after the deadline will be considered on a case-by-case basis.
  • ASH reserves the right to withdraw abstracts that are in violation of the Society’s policies and guidelines, such as those that have been previously published or presented, have been deemed scientifically unsound, or have been found to include inaccurate data, etc.
  • If you indicated at submission that you wish to withdraw your abstract if it is not accepted for either oral or poster presentation, your abstract will be automatically withdrawn, and you will be notified; you do not need to request withdrawal.

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Publication in the Abstracts Issue of Blood

  • All abstracts accepted for presentation (plenary, oral, or poster) at the ASH annual meeting are published in a special print and online supplemental issue of Blood, ASH's official journal. This special print issue of Blood is mailed to all ASH members in good standing, non-member Blood subscribers, and non-members in North America who pre-register for the ASH annual meeting. (International meeting registrants must pick up their books on site.)
  • Abstracts not selected for presentation will be published online only on the Blood Abstracts website http://bloodjournal.hematologylibrary.org/misc/ASH_Meeting_Abstracts_Info.dtl. If an author does not want the abstract published if it is not accepted for presentation, he/she must indicate that during submission.
  • ASH reserves the right not to publish abstracts that do not fit into any of the review categories, abstracts that inappropriately promote commercial interests, abstracts that are judged unethical, poorly written, or scientifically unsound, or abstracts considered inappropriate for publication for other reasons as determined by the Program Committee.

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Late-Breaking Abstracts

Recognizing that the results of some top-quality research may not be available by the general abstract submission deadline, the ASH Program Committee offers an option for late-breaking abstracts. The late-breaking abstracts submission site will open October 21 and will close October 28, 2010. Late-breaking abstracts will undergo regular peer-review evaluation. Only six abstracts will be accepted and presented in the Late-Breaking Abstracts session on Tuesday, December 7. The accepted abstracts will be published online only at http://bloodjournal.hematologylibrary.org/misc/ASH_Meeting_Abstracts_Info.dtl, and will not be part of the special Abstract supplemental issue of Blood. Late-breaking abstracts not accepted for presentation will not be published in any form.

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Abstract Submission Policies

Conflict-of-Interest Disclosure Policy

  • ASH is committed to ensuring the integrity of its scientific, educational, and research programs. The ASH Conflict-of-Interest Policy requires disclosure of any financial or other interest that might be construed as resulting in an actual, potential, or apparent conflict.
  • ASH abides by rules formulated by the Accreditation Council for Continuing Medical Education (ACCME) that require that you disclose any relevant financial relationship you or your spouse/partner have had within the past 24 months. For this purpose, “relevant financial relationships” are those from which you have received or may receive financial benefit and which are related to the CME content.
  • As a continuing medical education (CME) provider accredited by the ACCME, ASH must ensure balance, independence, objectivity, and scientific rigor in all presentations at the ASH annual meeting.
  • By completing this section of the online abstract submission, you agree that you have read the ASH Conflict-of-Interest Policy and that you understand and support its intent.
  • This policy is not intended to prevent a presentation; it is merely intended to openly identify potential conflicts so that audience members may form their own judgments about the presentation with a full disclosure of the facts.
  • Appropriate disclosure will be stated in the special abstract issue of Blood (the Abstracts issue). Abstracts will not be considered for the program without completion of disclosure information for all of the authors.

Author Responsibility Regarding Conflict-of-Interest Disclosure

  • The presenting author is responsible for obtaining disclosure information from all co-authors.
  • All authors and co-authors are required to provide any relevant information concerning personal or professional circumstances and relationships that might reasonably be expected to affect the author’s view on the presentation.
  • This includes relationships with pharmaceutical companies, biomedical device manufacturers, or other companies whose products or services are related to the subject matter of the presentation topic. If no relevant relationships exist, this must be stated as well.

When to Disclose

Please disclose any relationships or circumstances that might affect or appear to affect the research presented. These relationships include you or any individual with whom you directly share income.

What to Disclose

You must disclose the relationship and state the name of the company for each of the following areas in which you maintain a relationship. Exact dollar amounts are not necessary. You will have the option to note that there is no information to disclose or to provide disclosure information pertinent to the abstract. Disclosed information pertinent to the abstract may include the following:

Relationships

  • Employment
  • Consultancy
  • Ownership interests (including stock options) in a start-up company, the stock of which is not publicly traded
  • Ownership interest (including stock options, but excluding indirect investments through mutual funds and the like) in a publicly traded company
  • Research funding
  • Honoraria directly received from an entity
  • Patents and royalties
  • Paid expert testimony
  • Membership on an entity’s board of directors, speakers bureau, or its advisory committees
  • Any other financial relationship

Off-Label Use

You will be required to note whether your presentation will include discussion of off-label use of products. If so, you must provide a brief explanation.

Other Requirements

During the disclosure submission process, you will also be required to indicate your compliance with the following:

  • If you are providing recommendations involving clinical medicine, these recommendations will be based on evidence that is accepted within the profession of medicine as adequate justification for their indication and contraindications in the care of patients. All scientific research referred to in support of a patient-care recommendation will conform to generally accepted standards of experimental design, data collection, and analysis.
  • The content of the information with which you are involved will promote quality in health care or advances in science and will not promote a specific proprietary or commercial interest. Content for this publication will be well-balanced and unbiased.
  • If you have been trained or utilized by a commercial entity or its agents as a speaker (e.g., participation in a speakers bureau) for any commercial interest, the promotional aspects of that work must not be included in the presentation in any way.

Embargo Policy

  • Abstracts submitted to the ASH annual meeting are embargoed from the time of submission.
  • For the research to be eligible for presentation at the ASH annual meeting, information contained in the abstract, as well as additional data and information to be presented about the research at the annual meeting, may not be made public before the abstract has been published/presented at the ASH annual meeting.
  • Prior to the embargo being lifted, the first author, co-authors, and sponsor of the abstract must not:
    • Publish the information or provide it to others who may publish it.
    • Release the research/study to news media, or
    • Use the information for trading in the securities of any issuer, or provide it to others who may use it for securities-trading purposes.
  • If the Embargo Policy is violated, the abstract may be withdrawn by ASH from presentation at the annual meeting and from publication in print and online.
  • More detailed information about the Society’s Embargo Policy is located on the ASH website.
  • Authors who have questions about the Embargo Policy may send inquiries to ASHabstracts@hematology.org.
  • Reporters who have questions about the Embargo Policy may contact ASH Communications Specialist Claire Gwayi-Chore at cgwayi-chore@hematology.org with any questions.

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How to Submit an Abstract

The abstract submission site is open June 14 - August 12, 2010.

All abstracts must be submitted by August 12, 2010, at 11:59 p.m., Pacific Daylight Time. Submissions that are incomplete by the deadline will be rejected.

  • Abstracts must be submitted online through the official online abstract submission system. E-mails and word processing files not submitted through the site will not be accepted.
  • There is a $60 non-refundable handling fee for submitting an abstract. Payment must be made by credit card; Visa, MasterCard, and American Express are accepted. Purchase orders and checks will not be accepted. The abstract submission fee does not include registration for the annual meeting; therefore, all authors planning to attend the ASH annual meeting must register for the meeting.
  • Electronic submission works optimally with Microsoft Internet Explorer 7+, Mozilla Firefox 3.0+, and Safari or MAC OS 10.4+. Netscape Navigator 7.7+ is supported, but its use is discouraged, since not all features are available. The online abstract submission system will provide links so that you can download free browser software.
  • If this is the first time that you are submitting an abstract for the ASH annual meeting, please be sure to check the box that indicates this during submission.
  • Once you have submitted the title page information, a draft of your abstract will be saved, and you will be able to return to edit and update it at any time. You will receive an e-mail providing a link to your submission.
  • Abstracts cannot be submitted and will not be reviewed without proper payment and completion of the “Submission Information” and “Disclosure” sections of the online abstract submission program.

Any technical questions regarding the submission process should be directed to ash@confex.com.

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Preparing an Abstract for submission

Contact Information

  • Your name, degree, institution, address, phone number, and e-mail address must be provided. As the corresponding author, you will receive all future correspondence from ASH.
  • The corresponding author should be the first author (presenter) of the abstract, unless otherwise noted during submission.
  • If none of the abstract authors is an ASH member, the program will ask you to provide the name of the ASH member who is sponsoring the abstract.

Co-Authors

  • Names of co-authors and institutions must be provided. The program will automatically place an asterisk (*) after the name of each non-member author. Changes will not be made to the spelling of authors’ names after the submission deadline; please proof your co-authors’ names carefully.

Copyright Policy

  • All authors must assign copyright of the abstract to ASH, unless one of the authors is a U.S. Federal employee (in such case, ASH does not hold copyright).

Abstract Title

  • The abstract title should be brief and clearly indicate the nature of the abstract.
  • The abstract title must be in title case. Capitalize all nouns, pronouns, adjectives, verbs, adverbs, and subordinate conjunctions (i.e., as, because, although). Except for the first word of the title, lowercase all articles, coordinate conjunctions (i.e., and, or, nor), and prepositions, regardless of length. Also, lowercase “to” when used as an infinitive.
  • Additionally, keep letters lowercase if the lowercase letters have a specific meaning, such as pH or NaCl.
  • Do not put a period at the end of the title.

Use of Product Names

  • Non-proprietary (generic/scientific) names should be used and should be lowercase.
  • If necessary, you may include a proprietary name in parentheses directly following the generic name after its first mention in the body of the abstract; the first letter of the name of a proprietary drug should be capitalized. ASH reserves the right to replace proprietary names with generic names to adhere to this policy.

Abbreviations

  • Use standard abbreviations. Place abbreviations in parentheses immediately after the first mention of a term or phrase; the abbreviation can then be used throughout the abstract.

Abstract Body, Tables, and Figures

  • Abstracts submitted for the ASH annual meeting are published in a special November 19, 2010, issue of Blood, the Journal of the American Society of Hematology. Abstracts will be typeset from the text submitted by the author without copyediting changes. It is the responsibility of the author to proof read the abstract carefully.
  • The entire body of the abstract, including text and tables, must not exceed 3,800 characters. Spaces are not included in this number; title, authors’ names, and affiliations are counted separately. Figures are not included in the character count.
  • There is no preference for structured (i.e., abstracts divided into sections using terms such as Introduction, Methods, Results, Conclusions, etc.) vs. unstructured abstracts.
  • Do not use bold type or underline formatting. Italic type is acceptable.
  • The text must be a single paragraph. Do not indent the first line.
  • Special Greek and mathematical symbols are available in a character map within the submission system.
  • Use numerals to indicate numbers, except when beginning sentences.
  • Simple tables and graphs may be included. The online abstract submission system provides step-by-step instructions on inserting tables and graphs in the preferred HTML format.
  • Figures should be uploaded with your abstract text. All figures should be at 300 dpi or better.
  • Any references should be noted as citations within the text and not as footnotes at the end.

Selection of Abstract Review Category

  • Please refer to the list of this year’s abstract review categories, which provides detailed descriptions of each category, to assist you in selecting the correct abstract classification.
  • Be sure to select the review category that best describes your abstract. Note that the abstract will be reviewed in the category you selected; there is no re-classification once submission has closed.

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Travel Awards

  • Undergraduate students, medical students, graduate students, resident physicians, and post-doctoral (MD or PhD) fellows who are both first author and presenter of the abstract are eligible for an ASH Travel Award.
  • ASH Travel Awards of $500 are granted to selected individuals in conjunction with the ASH annual meeting. Travel Awards are granted on the basis of merit to those who have demonstrated a financial need for travel support. They are intended to help defray annual meeting travel costs for abstract presenters who demonstrate a need for financial assistance.

How to Apply for a Travel Award

  • To apply for a Travel Award, you must first identify yourself as a hematologist in training (i.e., undergraduate, medical, or graduate student; resident physician; or post doctoral fellow) in the online abstract submission system.
  • To complete your Travel Award application, you must submit a letter requesting travel support from your training program director.
  • Your letter must be typed on your institution’s letterhead, signed by your training program director, saved in PDF format, and uploaded to the online abstract submission system.
  • Please include the following items in your letter:
    • Identify the need for a travel award
    • Your abstract title
    • Author name (this is the presenting/contact author)
    • Filename of the electronic submission

If applying for a Travel Award, authors must upload the required request letter into the program in order to complete the abstract submission. The deadline for receipt of the Travel Award request letter is, therefore, August 12, 2010. There will be no exceptions.

Travel Award Notifications

Travel Award applicants will be notified as to whether or not they received an award by late October 2010. Please contact ASH Headquarters if you have not heard about the status of your award by November 1, 2010.

How to Receive Your Travel Award

Travel Awards must be picked up on site at the ASH annual meeting in Orlando, Florida. Recipients must go to the Membership Desk in the Orange County Convention Center, provide a photo ID, and sign for receipt of the award. Checks that are not picked up on site at the ASH annual meeting will be voided.

Authors who do not have the required Travel Award request letter may still be considered for the ASH Merit Award, as long as the box indicating that the author is a hematologist in training is checked during submission.

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Merit Awards

  • ASH Merit Awards are granted without regard to need in the categories of undergraduate student, medical student, graduate student, resident physician, and post-doctoral (MD or PhD) fellow to the first author and presenter of the highest scoring abstract in each scientific category.
  • Merit Award winners receive a $500 honorarium plus annual meeting travel reimbursement (coach airfare and two nights' hotel accommodations).

How to Apply for a Merit Award

An individual who self-identifies as a hematologist in training in the abstract submission system is automatically considered for a Merit Award. The Merit Awards are not based on financial need; therefore, no letter is required in order to apply.

Merit Award Notifications

Merit Award applicants will be notified as to whether they have received an award by late October 2010. Please contact ASH Headquarters if you have not heard about the status of your award by November 1, 2010.

How to Receive Your Merit Award

Merit Awards will be distributed to winners at the time of their respective presentations during the ASH annual meeting in Orlando, Florida.

Special ASH Merit Award!

Mary Rodes Gibson Memorial Award in Hemostasis & Thrombosis

  • This annual award shall be granted to a trainee (undergraduate student, medical student, graduate student, resident physician, or post-doctoral [MD or PhD] fellow) who is the first author and presenter of the highest scoring abstract submitted in the field of hemostasis and thrombosis.
  • The recipient of the award shall receive a $5,000 grant and a $1,000 allowance for travel and expenses to the ASH annual meeting.
  • This annual award is made possible by the Mary Rodes Gibson Hemostasis-Thrombosis Foundation to continue the legacy of Mary Rodes Gibson, who suffered from severe, type 3 von Willebrand disease.

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Electronic Signature

Completion of all required disclosure information in the online abstract submission system serves as an agreement and is accepted in lieu of a faxed signature. It certifies the ASH abstract submitter's understanding of the rules for participation contained in the online abstract submission program and affirms that:

  1. All authors approve of submitting this work for presentation and publication;
  2. The author(s) transfer(s) all copyright ownership of the named abstract to the American Society of Hematology (except when one or more authors are U.S. Government employees);
  3. All authors have read the ASH Abstract Conflict-of-Interest Policy and have acted in accordance with that policy;
  4. The author(s) agree(s) to materially confine the presentation to information in the abstract, if accepted for presentation. If an author has more than one abstract accepted, each presentation will be materially confined to the information in the abstract selected for the specific session;
  5. The presenting author will be available to present the abstract if selected for the program. The author(s) will immediately notify ASH if the presenting author must be changed; and
  6. The data in the abstract are not expected to be published, either online or in print, by August 18, 2010, nor will they be materially presented at another major meeting or at any Friday Satellite Symposium prior to the ASH annual meeting.

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Sample Abstract

A sample abstract is provided for your reference below.

RAD001, An Inhibitor of mTOR, Shows Clinical Activity in a Phase I/II Study in Patients with Primary Myelofibrosis (PMF) and Post Polycythemia Vera/Essential Thrombocythemia Myelofibrosis (PPV/PET MF). Alessandro M. Vannucchi, MD1, Paola Guglielmelli, MD*,1, Elisabetta Gattoni, MD*,2, Elisabetta Antonioli, MD*,1, Costanza Bogani, PhD*,1, Lorenzo Tozzi, BSc*,1, Lisa Pieri, MD*,1, Alberto Bosi, MD*,1, Alessandro Rambaldi, MD3, Tiziano Barbui, MD*,3 and Giovanni Barosi, MD*,2, 1 Hematology, Universitá degli Studi di Firenze, Firenze, Italy, 2 Unit Clin Epidemiol & Centre for the Study of Myelofibrosis, IRCCS Policlinico San Matteo Foundation, Pavia, Italy, 3 Hematology, Ospedali Riuniti di Bergamo, Bergamo, Italy

Constitutive and enhanced activation of JAK/STAT pathway isfound in patients (pts) with myeloproliferative neoplasms (MPN),particularly but not exclusively in those with mutation in theJanus tyrosine kinase 2 gene (JAK2V617F) or the MPL gene (MPLW515L/K/A).Activation of the Erk and PI3K/Akt pathways have also been describedin cells harboring the JAK2V617F mutation. In in-vitro studiesperformed in our laboratory (AM Vannucchi et al, ASH 2009, abstractsubmitted) we observed that RAD001, a specific inhibitor ofthe serine/threonine kinase mTOR (mammalian target of rapamycin)that is a downstream target of PI3K, remarkably inhibited proliferationof JAK2V617F mutant cell lines and reduced the clonogenic activityof CD34+ cells from MPN pts. The PI3K/Akt/mTOR pathway has becomean important focus for cancer therapy, and mTOR inhibitors havedemonstrated activity against solid tumors and lymphomas. Aphase I/II trial of RAD001 is being conducted in pts with PMFand PPV/PET MF, including JAK2V617F or MPL mutated as well aswild-type pts with intermediate/high risk score according toLille criteria. Diagnosis of PMF and of PPV/PET-MF was madefollowing the WHO and International Working Group for MyelofibrosisResearch and Treatment (IWG-MRT) criteria, respectively. PhaseI is a dose escalation of RAD001 involving 3 sequential cohortsof 3 pts each at 5.0, 7.5, and 10 mg daily, orally, for 3 months,to define maximum tolerated dose (MTD). Phase II involves theuse of MTD for 4 months, according to a two-stage Simon design,aimed at assessing efficacy of the drug. Responses are evaluatedusing the EUMNET criteria for treatment response in myelofibrosis(Barosi G et al, Blood 2005). At the time of writing, 8 of 9pts included in the Phase I have completed the 3-month studyperiod without dose-limiting toxicities or other significantadverse events; the ninth pt at 10 mg is completing the lastmonth of treatment. There were seven pts with PMF and 2 withPPV-MF. Main toxicities were represented by grade 2 mouth ulcersin 3 pts and transient grade 1/2 hypertrigliceridemia in 5 pts,that occurred irrespective of the dose. One pt at 10 mg presentedtransient muscular weakness and arthralgia. The initial doseof 5.0 mg resulted in a reduction in spleen size consistentwith a complete response (CR) in one pt (from 4 to 0 cm belowthe left costal margin) and partial responses (PR) in two pts(from 10 and 13 cm to 1 and 9 cm, respectively). In the 7.5mg cohort, there were one CR (from 4 to 0 cm) and one PR (from16 to 8 cm) in splenomegaly; none of the 2 pts at 10 mg hada response in splenomegaly. In 1 pt CR in splenomegaly was maintainedat +4 mo after suspension of drug while in the other respondingpts there was a progressive re-enlargement of the spleen to baselinelevel. A complete resolution of systemic symptoms was achievedin 2 of 3 pts while aquagenic pruritus disappeared in all the5 pts who complained of it. Overall, there were 2 major and3 moderate responses, while 3 pts did not show a measurableresponse. At the end of therapy, the absolute count of circulatingCD34+ cells had decreased in all but one pt of a median valueof 39% from baseline (range 16% to 72%). There was no significantdecrease in the burden of granulocyte V617F allele at the endof treatment in the 8 mutated pts. Single-colony JAK2V617F genotypingwas performed in 4 pts, 2 each from the 5.0 and 7.5 mg cohort.In one patient receiving 7.5 mg the percentage of homozygousBFU-E and CFU-GM decreased from 14% and 45%, respectively, atbaseline to 0 and 18% at the end of therapy; in the other 3pts changes were minimal. In conclusion, initial results showedrapid reduction of splenomegaly and symptomatic improvement,without relevant toxicity, in pts with myelofibrosis receivingRAD001. The drug also reduced the circulating CD34+ cell countand V617F-homozygous progenitor cells appeared to be decreasingat least in some patients, although the allelic burden measuredin granulocytes did not modify. Updated results on current patientsand future patients enrolled in Phase II will be presented atthe meeting.

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