JAK inhibitors for myeloproliferative neoplasms: Clarifying facts from myths

Research output: Contribution to journalArticle

115 Citations (Scopus)

Abstract

On November 16, 2011, the Food and Drug Administration approved ruxolitinib (a JAK1 and JAK2 inhibitor) for use in the treatment of high and intermediate risk myelofibrosis. This is welcome news for those patients in whom such therapy is indicated and treatment benefit outweighs attendant risk. The question is who are these patients, what should they expect in terms of both short-term effects and long-term impact, and why would they choose ruxolitinib over other JAK inhibitors that are freely available for use in a research setting. Ruxolitinib and most other JAK inhibitors exert a salutary effect on constitutional symptoms and splenomegaly but have yet to produce histopathologic or cytogenetic remissions, reverse bone marrow fibrosis, or improve survival over best supportive care. Furthermore, the palliative value of JAK inhibitors is diminished by notable side effects, including anemia, thrombocytopenia, gastrointestinal disturbances, metabolic abnormalities, peripheral neuropathy, and hyperacute relapse of symptoms during treatment discontinuation. Therefore, risk-benefit balance favors use of currently available JAK inhibitors in only a select group of patients with myelofibrosis, and their potential value in polycythemia vera, outside of special circumstances (eg, intractable pruritus), is undermined by the absence of evidence for a disease-modifying effect and presence of arguably superior alternatives.

Original languageEnglish (US)
Pages (from-to)2721-2730
Number of pages10
JournalBlood
Volume119
Issue number12
DOIs
StatePublished - Mar 22 2012

Fingerprint

Primary Myelofibrosis
Neoplasms
Polycythemia Vera
Splenomegaly
Bone
Peripheral Nervous System Diseases
United States Food and Drug Administration
Therapeutics
Pruritus
Cytogenetics
Thrombocytopenia
Anemia
Recurrence
Survival
INCB018424
Research

ASJC Scopus subject areas

  • Hematology
  • Biochemistry
  • Cell Biology
  • Immunology

Cite this

JAK inhibitors for myeloproliferative neoplasms : Clarifying facts from myths. / Tefferi, Ayalew.

In: Blood, Vol. 119, No. 12, 22.03.2012, p. 2721-2730.

Research output: Contribution to journalArticle

@article{2c1bb87539b24af291f4c0de21aa9b67,
title = "JAK inhibitors for myeloproliferative neoplasms: Clarifying facts from myths",
abstract = "On November 16, 2011, the Food and Drug Administration approved ruxolitinib (a JAK1 and JAK2 inhibitor) for use in the treatment of high and intermediate risk myelofibrosis. This is welcome news for those patients in whom such therapy is indicated and treatment benefit outweighs attendant risk. The question is who are these patients, what should they expect in terms of both short-term effects and long-term impact, and why would they choose ruxolitinib over other JAK inhibitors that are freely available for use in a research setting. Ruxolitinib and most other JAK inhibitors exert a salutary effect on constitutional symptoms and splenomegaly but have yet to produce histopathologic or cytogenetic remissions, reverse bone marrow fibrosis, or improve survival over best supportive care. Furthermore, the palliative value of JAK inhibitors is diminished by notable side effects, including anemia, thrombocytopenia, gastrointestinal disturbances, metabolic abnormalities, peripheral neuropathy, and hyperacute relapse of symptoms during treatment discontinuation. Therefore, risk-benefit balance favors use of currently available JAK inhibitors in only a select group of patients with myelofibrosis, and their potential value in polycythemia vera, outside of special circumstances (eg, intractable pruritus), is undermined by the absence of evidence for a disease-modifying effect and presence of arguably superior alternatives.",
author = "Ayalew Tefferi",
year = "2012",
month = "3",
day = "22",
doi = "10.1182/blood-2011-11-395228",
language = "English (US)",
volume = "119",
pages = "2721--2730",
journal = "Blood",
issn = "0006-4971",
publisher = "American Society of Hematology",
number = "12",

}

TY - JOUR

T1 - JAK inhibitors for myeloproliferative neoplasms

T2 - Clarifying facts from myths

AU - Tefferi, Ayalew

PY - 2012/3/22

Y1 - 2012/3/22

N2 - On November 16, 2011, the Food and Drug Administration approved ruxolitinib (a JAK1 and JAK2 inhibitor) for use in the treatment of high and intermediate risk myelofibrosis. This is welcome news for those patients in whom such therapy is indicated and treatment benefit outweighs attendant risk. The question is who are these patients, what should they expect in terms of both short-term effects and long-term impact, and why would they choose ruxolitinib over other JAK inhibitors that are freely available for use in a research setting. Ruxolitinib and most other JAK inhibitors exert a salutary effect on constitutional symptoms and splenomegaly but have yet to produce histopathologic or cytogenetic remissions, reverse bone marrow fibrosis, or improve survival over best supportive care. Furthermore, the palliative value of JAK inhibitors is diminished by notable side effects, including anemia, thrombocytopenia, gastrointestinal disturbances, metabolic abnormalities, peripheral neuropathy, and hyperacute relapse of symptoms during treatment discontinuation. Therefore, risk-benefit balance favors use of currently available JAK inhibitors in only a select group of patients with myelofibrosis, and their potential value in polycythemia vera, outside of special circumstances (eg, intractable pruritus), is undermined by the absence of evidence for a disease-modifying effect and presence of arguably superior alternatives.

AB - On November 16, 2011, the Food and Drug Administration approved ruxolitinib (a JAK1 and JAK2 inhibitor) for use in the treatment of high and intermediate risk myelofibrosis. This is welcome news for those patients in whom such therapy is indicated and treatment benefit outweighs attendant risk. The question is who are these patients, what should they expect in terms of both short-term effects and long-term impact, and why would they choose ruxolitinib over other JAK inhibitors that are freely available for use in a research setting. Ruxolitinib and most other JAK inhibitors exert a salutary effect on constitutional symptoms and splenomegaly but have yet to produce histopathologic or cytogenetic remissions, reverse bone marrow fibrosis, or improve survival over best supportive care. Furthermore, the palliative value of JAK inhibitors is diminished by notable side effects, including anemia, thrombocytopenia, gastrointestinal disturbances, metabolic abnormalities, peripheral neuropathy, and hyperacute relapse of symptoms during treatment discontinuation. Therefore, risk-benefit balance favors use of currently available JAK inhibitors in only a select group of patients with myelofibrosis, and their potential value in polycythemia vera, outside of special circumstances (eg, intractable pruritus), is undermined by the absence of evidence for a disease-modifying effect and presence of arguably superior alternatives.

UR - http://www.scopus.com/inward/record.url?scp=84858855487&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84858855487&partnerID=8YFLogxK

U2 - 10.1182/blood-2011-11-395228

DO - 10.1182/blood-2011-11-395228

M3 - Article

C2 - 22279053

AN - SCOPUS:84858855487

VL - 119

SP - 2721

EP - 2730

JO - Blood

JF - Blood

SN - 0006-4971

IS - 12

ER -