Valentina Bellani, Barbara Mora, Alessandra Iurlo, Francesco Passamonti
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引用次数: 0
Abstract
Calreticulin (CALR) is a chaperone protein that plays a crucial role in protein folding quality control and calcium homeostasis. Mutations in CALR result in a mutated protein lacking key calcium-binding sites and the KDEL sequence, leading to a constitutive activation of the MPL-JAK2-STAT5 pathway, which is involved in the pathogenesis of essential thrombocythemia (ET) and primary myelofibrosis (PMF). Despite advancements in understanding the role of CALR mutations, current therapeutic strategies remain focused on managing symptoms and complications, with allogeneic stem cell transplantation (alloHSCT) as the only curative option. Emerging research is exploring novel immunotherapeutic approaches targeting mutant CALR, including the development of anti-CALR antibodies and T-cell receptor-mediated therapies, offering potential new avenues for treatment in CALR-mutated MPNs. In this review, we will discuss on the role of CALR in MPNs, focusing on its biological structure and its implications on the prognosis and treatment of essential thrombocythemia and primary myelofibrosis.
期刊介绍：
Leukemia & Lymphoma in its fourth decade continues to provide an international forum for publication of high quality clinical, translational, and basic science research, and original observations relating to all aspects of hematological malignancies. The scope ranges from clinical and clinico-pathological investigations to fundamental research in disease biology, mechanisms of action of novel agents, development of combination chemotherapy, pharmacology and pharmacogenomics as well as ethics and epidemiology. Submissions of unique clinical observations or confirmatory studies are considered and published as Letters to the Editor
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