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Make a fundraising page JustGiving The CML Support Group is an online patient support group for chronic myeloid leukaemia patients, their families and carers. The website has been running since April 2000 and has a lively discussion forum as well as information regarding current therapies for CML.

The impact of healthcare settings on survival time of patients with chronic myeloid leukemia Michael Lauseker

Michael Lauseker1,Joerg Hasford1, Markus Pfirrmann1, and Rüdiger Hehlmann2

Key points

* Chronic myeloid leukemia patients enjoyed superior survival chances when treated in teaching hospitals.

* Less experience of a treatment center with chronic myeloid leukemia patients did not affect the patient's survival chances.

With the introduction of tyrosine kinase inhibitors the treatment of chronic myeloid leukemia (CML) patients has migrated extensively to municipal hospitals (MH) and office-based physicians (OBP). Thus we wanted to check whether the health care setting has an impact on outcome. Based on 1491 patients of the German CML study IV, we compared the outcomes of patients from teaching hospitals (TH) to those from MH and OBP. Adjusting for age, EUTOS score, Karnofsky performance status, year of diagnosis and experience with chronic myeloid leukemia (CML), a significant survival advantage for TH patients (HR: 0.632 resp. 0.609) was found. In particular, when treated in TH, patients with blast crisis showed a superior outcome (2 year survival rate: 47.7% vs. 22.3% vs. 25.0%). As such an impact of the health care setting on the outcome of CML patients has not been reported before these findings need confirmation by other study groups.
This study is registered at, identifier: NCT00055874.

Submitted November 20, 2013.
Accepted March 5, 2014.
Copyright © 2014 American Society of Hematology

Long-term outcome with dasatinib after imatinib failure in chronic-phase chronic myeloid leukemia: follow-up of phase 3 study

Neil P. Shah1,*, François Guilhot2, Jorge E. Cortes3, Charles A. Schiffer4, Philipp le Coutre5, Tim H. Brümmendorf6, Hagop M. Kantarjian3, Andreas Hochhaus7, Philippe Rousselot8, Hesham Mohamed9, Diane Healey10, Michael Cunningham10, and Giuseppe Saglio11
+ Author Affiliations

Key points

* Imatinib-resistant/-intolerant patients with chronic myeloid leukemia in chronic phase can experience long-term benefit with dasatinib.

* Early (3- and 6-month) molecular and cytogenetic responses were associated with improved progression-free survival and overall survival.

Chronic Myeloid Leukemia: 2013 American Society of Hematology Annual Meeting*

*Clinical Care Options: Oncology.
In this Expert Analysis, Jorge E. Cortes, MD, and Michael J. Mauro, MD, provide an in-depth review of the most clinically relevant new data presented at the 2013 American Society of Hematology meeting on current and emerging therapies for the treatment of chronic myeloid leukemia.

Avillion Group Partners with Pfizer to Co-develop BOSULIF® (bosutinib) as First-Line Treatment for Patients with Chronic Myelogenous Leukemia

London, UK, January 09, 2014 --

The Avillion Group (Avillion), a co-developer of late-stage clinical assets, announced today that it has entered into an exclusive collaborative development agreement with Pfizer Inc. to conduct a global Phase 3 clinical trial of Pfizer’s BOSULIF® (bosutinib).

The trial, which will be conducted across multiple sites in the United States, Asia and Europe, will evaluate BOSULIF, administered at a starting dose level of 400 mg daily, as a first-line treatment for patients with chronic phase Philadelphia chromosome positive (Ph+) chronic myelogenous leukemia (CML).

Under the terms of the agreement, Avillion will provide the funding for and will conduct the trial to generate the clinical data necessary to potentially support a registration dossier for marketing authorization of BOSULIF by regulatory authorities for an indication as first-line treatment of patients with chronic phase Ph+ CML. If approved for this indication, Avillion will be eligible to receive milestone payments from Pfizer upon regulatory approval of the drug. Pfizer will retain all rights to commercialize BOSULIF globally.

"We are delighted to announce Avillion’s agreement with Pfizer, a global leader in the biopharmaceutical industry, and we look forward to working with them with the goal of advancing the development of BOSULIF and expanding its availability to a broader range of CML patients,” said Lewis Cameron, CEO of Avillion. “Avillion offers pharmaceutical and biotech companies a compelling option to partner late-stage drug development projects. We have an experienced team focused on global drug development and regulatory approval, with the capability to optimise contract research organization (CRO) management.”

European LeukemiaNet recommendations for the management of chronic myeloid leukemia: 2013

Michele Baccarani1, Michael W. Deininger2, Gianantonio Rosti3, Andreas Hochhaus4, Simona Soverini3, Jane F. Apperley5, Francisco Cervantes6, Richard E. Clark7, Jorge E. Cortes8, François Guilhot9, Henrik Hjorth-Hansen10, Timothy P. Hughes11, Hagop M. Kantarjian8, Dong-Wook Kim12, Richard A. Larson13, Jeffrey H. Lipton14, François-Xavier Mahon15, Giovanni Martinelli3, Jiri Mayer16, Martin C. Müller17, Dietger Niederwieser18, Fabrizio Pane19, Jerald P. Radich20, Philippe Rousselot21, Giuseppe Saglio22, Susanne Saußele17, Charles Schiffer23, Richard Silver24, Bengt Simonsson25, Juan-Luis Steegmann26, John M. Goldman27, and Rüdiger Hehlmann17

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ARIAD Announces the Commercial Availability of Iclusig (Ponatinib) for Patients with Refractory Philadelphia-Positive Leukemias in the U.S.

17th January 2014
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA) today announced the commercial availability of Iclusig® (ponatinib) for adult patients with refractory chronic myeloid leukemia and Philadelphia-chromosome positive acute lymphoblastic leukemia in the United States. ARIAD has begun shipping Iclusig to Biologics, Inc., its exclusive specialty pharmacy, which is now filling prescriptions from physicians and distributing the cancer medicine to patients.

We are pleased to again have Iclusig commercially available to patients in the United States, stated Marty J. Duvall, executive vice president and chief commercial officer for ARIAD. Iclusig is now in our distribution channel with Biologics, and our dedicated sales force will begin promoting Iclusig immediately. We are highly confident in our commercial launch of Iclusig and look forward to reporting on our progress on a quarterly basis.

Last month, the U.S. Food and Drug Administration (FDA) approved revised U.S. Prescribing Information (USPI) and a communications Risk Evaluation and Mitigation Strategy (REMS) for Iclusig that allowed for the immediate resumption of its marketing and commercial distribution. The USPI includes a revised indication statement and boxed warning, updated safety information and recommendations regarding dosing considerations for prescribers. Iclusig is now indicated for the treatment of adult patients with:

T315I-positive chronic myeloid leukemia (chronic phase, accelerated phase, or blast phase) or T315I-positive Philadelphia chromosome positive (Ph+) acute lymphoblastic leukemia, or
Chronic phase, accelerated phase, or blast phase chronic myeloid leukemia or Ph+ acute lymphoblastic leukemia for whom no other tyrosine-kinase inhibitor therapy is indicated.
The starting dose of Iclusig remains 45 mg daily. more

Bone Marrow Microenvironment Could Offer Future Target in CML

December 31, 2013 | Chronic Myeloid Leukemia, Hematologic Malignancies, Leukemia & Lymphoma
By Dave Levitan

A small, hypolobated megakaryocyte in a bone marrow aspirate, typical ...
Modulation of the bone marrow microenvironment with parathyroid hormone (PTH) may be a feasible way to dramatically reduce counts of leukemia stem cells in chronic myeloid leukemia (CML) patients, according to new research in mice. Reducing leukemia stem cells (LSCs) is a required step on the path to a true cure for the disease.

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A common novel splice variant of SLC22A1 (OCT1) is associated with impaired responses to imatinib in patients with chronic myeloid leukaemia.

Br J Haematol. 2013 Dec;163(5):631-9. doi: 10.1111/bjh.12591. Epub 2013 Oct 10.

Grinfeld J, Gerrard G, Alikian M, Alonso-Dominguez J, Ale S, Valgañon M, Nteliopoulos G, White D, Marin D, Hedgley C, O'Brien S, Clark R, Goldman JM, Milojkovic D, Apperley JF, Foroni L.


Approximately one-third of patients with chronic myeloid leukaemia will fail to achieve or maintain responses to imatinib. Changes in solute carrier family 22 (organic cation transporter), member 1 (SLC22A1, also termed OCT1), the main transporter for imatinib, have been proposed as a possible predictive factor. We analysed SLC22A1 mRNA levels and single nucleotide polymorphisms (SNPs) located in exon 7 in 153 diagnostic whole blood samples from two patient cohorts. The level of SLC22A1 expression did not significantly correlate with imatinib failure or achievement of molecular remission. The SNP 408V>M (g.1222G>A) was present in 65% of patients and was associated in all cases with an eight base-pair insertion (8(+) allele) at the 3' end of exon 7. The latter generates an alternative splice site, leading to a premature stop codon. M420del was found in 33% of patients and never in cis with 8(+) (the 3(-) allele). Significantly longer times to 1% and 0·1% molecular responses (by quantitative reverse transcription polymerase chain reaction) were seen in patients with 8(+) 8(+) or 8(+) N compared to those with the remaining four genotypes (N = no insertion or deletion). Patients lacking 8(+) and 3(-) (NN, 18%) showed the best outcomes overall. Thus, while SLC22A1 expression does not appear to affect response, alterations in its splicing or amino acid sequence may do so. © 2013 John Wiley & Sons Ltd.

chronic myeloid leukaemia, drug resistance, prognostic factors, tyrosine kinases

Penn Medicine Team Reports Findings from Research Study

Results of study: Personalized Cellular Therapy CTL019

NEW ORLEANS — Three and a half years after beginning a clinical trial which demonstrated the first successful and sustained use of genetically engineered T cells to fight leukemia, a research team from the Perelman School of Medicine at the University of Pennsylvania and the Children’s Hospital of Philadelphia will today announce the latest results of studies involving both adults and children with advanced blood cancers that have failed to respond to standard therapies. The findings from the first 59 patients who received this investigational, personalized cellular therapy, known as CTL019, will be presented during the American Society of Hematology’s Annual Meeting and Exposition in New Orleans.

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