Treatment of hereditary autoinflammatory diseases

Nienke M Ter Haar; Joost Frenkel

doi:10.1097/BOR.0000000000000059

Treatment of hereditary autoinflammatory diseases

Nienke M Ter Haar
, Joost Frenkel^*

^*Corresponding author for this work

Research output: Contribution to journal › Review article › peer-review

Abstract

PURPOSE OF REVIEW: The purpose of this review is to summarize recent advances in the treatment of the hereditary autoinflammatory diseases, focusing on Familial Mediterranean fever (FMF), tumor necrosis factor receptor-associated periodic syndrome (TRAPS), cryopyrin-associated periodic syndromes (CAPS) and mevalonate kinase deficiency (MKD). We discuss recently published studies and their implications for current patient care and future clinical research.

RECENT FINDINGS: Interleukin (IL)-1 blockade is effective in most autoinflammatory conditions. Younger patients require a higher dose per kg of body weight. In FMF, colchicine remains the treatment of choice. Single daily dosing appears adequate. When colchicine fails, IL-1 blockade is effective. In CAPS, the beneficial effect of IL-1 blockade is sustained, and side-effects are limited. There is no evidence that one IL-1 blocker is superior to the other. In TRAPS and MKD, IL-1 blockade appears effective. Some patients have sufficient suppression of inflammatory symptoms with NSAIDs or corticosteroids.

SUMMARY: Apart from CAPS and FMF, therapy of autoinflammatory diseases is based on small and retrospective studies. IL-1 blockade appears effective, but larger prospective trials are needed, especially in MKD, TRAPS and colchicine-resistant FMF patients.

VIDEO ABSTRACT:

Original language	English
Pages (from-to)	252-8
Number of pages	7
Journal	Current Opinion in Rheumatology
Volume	26
Issue number	3
DOIs	https://doi.org/10.1097/BOR.0000000000000059
Publication status	Published - 2014

Keywords

Biological Products
Colchicine
Cryopyrin-Associated Periodic Syndromes
Familial Mediterranean Fever
Hereditary Autoinflammatory Diseases
Humans
Interleukin-1
Mevalonate Kinase Deficiency
Severity of Illness Index
Journal Article
Research Support, Non-U.S. Gov't
Review

Access to Document

10.1097/BOR.0000000000000059

1 Comment/Letter to the editor

Erratum: Treatment of hereditary autoinflammatory diseases (Current Opinion in Rheumatology (2014) 26 (252-258))
Ter Haar, N. M. & Frenkel, J., 1 Jan 2014, In: Current Opinion in Rheumatology. 26, 4, 1 p.
Research output: Contribution to journal › Comment/Letter to the editor › Academic › peer-review

Cite this

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title = "Treatment of hereditary autoinflammatory diseases",

abstract = "PURPOSE OF REVIEW: The purpose of this review is to summarize recent advances in the treatment of the hereditary autoinflammatory diseases, focusing on Familial Mediterranean fever (FMF), tumor necrosis factor receptor-associated periodic syndrome (TRAPS), cryopyrin-associated periodic syndromes (CAPS) and mevalonate kinase deficiency (MKD). We discuss recently published studies and their implications for current patient care and future clinical research.RECENT FINDINGS: Interleukin (IL)-1 blockade is effective in most autoinflammatory conditions. Younger patients require a higher dose per kg of body weight. In FMF, colchicine remains the treatment of choice. Single daily dosing appears adequate. When colchicine fails, IL-1 blockade is effective. In CAPS, the beneficial effect of IL-1 blockade is sustained, and side-effects are limited. There is no evidence that one IL-1 blocker is superior to the other. In TRAPS and MKD, IL-1 blockade appears effective. Some patients have sufficient suppression of inflammatory symptoms with NSAIDs or corticosteroids.SUMMARY: Apart from CAPS and FMF, therapy of autoinflammatory diseases is based on small and retrospective studies. IL-1 blockade appears effective, but larger prospective trials are needed, especially in MKD, TRAPS and colchicine-resistant FMF patients.VIDEO ABSTRACT: ",

keywords = "Biological Products, Colchicine, Cryopyrin-Associated Periodic Syndromes, Familial Mediterranean Fever, Hereditary Autoinflammatory Diseases, Humans, Interleukin-1, Mevalonate Kinase Deficiency, Severity of Illness Index, Journal Article, Research Support, Non-U.S. Gov't, Review",

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year = "2014",

doi = "10.1097/BOR.0000000000000059",

language = "English",

volume = "26",

pages = "252--8",

journal = "Current Opinion in Rheumatology",

issn = "1040-8711",

publisher = "Lippincott Williams \& Wilkins",

number = "3",

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TY - JOUR

T1 - Treatment of hereditary autoinflammatory diseases

AU - Ter Haar, Nienke M

AU - Frenkel, Joost

PY - 2014

Y1 - 2014

N2 - PURPOSE OF REVIEW: The purpose of this review is to summarize recent advances in the treatment of the hereditary autoinflammatory diseases, focusing on Familial Mediterranean fever (FMF), tumor necrosis factor receptor-associated periodic syndrome (TRAPS), cryopyrin-associated periodic syndromes (CAPS) and mevalonate kinase deficiency (MKD). We discuss recently published studies and their implications for current patient care and future clinical research.RECENT FINDINGS: Interleukin (IL)-1 blockade is effective in most autoinflammatory conditions. Younger patients require a higher dose per kg of body weight. In FMF, colchicine remains the treatment of choice. Single daily dosing appears adequate. When colchicine fails, IL-1 blockade is effective. In CAPS, the beneficial effect of IL-1 blockade is sustained, and side-effects are limited. There is no evidence that one IL-1 blocker is superior to the other. In TRAPS and MKD, IL-1 blockade appears effective. Some patients have sufficient suppression of inflammatory symptoms with NSAIDs or corticosteroids.SUMMARY: Apart from CAPS and FMF, therapy of autoinflammatory diseases is based on small and retrospective studies. IL-1 blockade appears effective, but larger prospective trials are needed, especially in MKD, TRAPS and colchicine-resistant FMF patients.VIDEO ABSTRACT:

AB - PURPOSE OF REVIEW: The purpose of this review is to summarize recent advances in the treatment of the hereditary autoinflammatory diseases, focusing on Familial Mediterranean fever (FMF), tumor necrosis factor receptor-associated periodic syndrome (TRAPS), cryopyrin-associated periodic syndromes (CAPS) and mevalonate kinase deficiency (MKD). We discuss recently published studies and their implications for current patient care and future clinical research.RECENT FINDINGS: Interleukin (IL)-1 blockade is effective in most autoinflammatory conditions. Younger patients require a higher dose per kg of body weight. In FMF, colchicine remains the treatment of choice. Single daily dosing appears adequate. When colchicine fails, IL-1 blockade is effective. In CAPS, the beneficial effect of IL-1 blockade is sustained, and side-effects are limited. There is no evidence that one IL-1 blocker is superior to the other. In TRAPS and MKD, IL-1 blockade appears effective. Some patients have sufficient suppression of inflammatory symptoms with NSAIDs or corticosteroids.SUMMARY: Apart from CAPS and FMF, therapy of autoinflammatory diseases is based on small and retrospective studies. IL-1 blockade appears effective, but larger prospective trials are needed, especially in MKD, TRAPS and colchicine-resistant FMF patients.VIDEO ABSTRACT:

KW - Biological Products

KW - Colchicine

KW - Cryopyrin-Associated Periodic Syndromes

KW - Familial Mediterranean Fever

KW - Hereditary Autoinflammatory Diseases

KW - Humans

KW - Interleukin-1

KW - Mevalonate Kinase Deficiency

KW - Severity of Illness Index

KW - Journal Article

KW - Research Support, Non-U.S. Gov't

KW - Review

U2 - 10.1097/BOR.0000000000000059

DO - 10.1097/BOR.0000000000000059

M3 - Review article

C2 - 24667287

SN - 1040-8711

VL - 26

SP - 252

EP - 258

JO - Current Opinion in Rheumatology

JF - Current Opinion in Rheumatology

IS - 3

ER -

Treatment of hereditary autoinflammatory diseases

Abstract

Keywords

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Research output

Erratum: Treatment of hereditary autoinflammatory diseases (Current Opinion in Rheumatology (2014) 26 (252-258))

Cite this