Scleromyxedema: role of high-dose melphalan with autologous stem cell transplantation

Donato, Michèle; Feasel, Adrienne M.; Weber, Donna M.; Prieto, Víctor G.; Giralt, Sergío; Champlin, Richard E.; Duvic, Madeleine

doi:10.1182/blood-2004-12-4870

Cited by 62 publications

(47 citation statements)

References 14 publications

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“…Thalidomide has been reported to be effective in resistant cases. PBSCT led to complete resolution of most cutaneous and systemic symptoms in some patients with scleromyxedema, 110 but its efficacy and safety need confirmation.…”

Section: Poems Syndromementioning

confidence: 99%

Dangerous small B-cell clones

Merlini

Stone

2006

Blood

394

280

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The detection of a monoclonal immunoglobulin in serum or urine usually raises concerns about the size of the underlying B-cell-derived clone and possible systemic effects caused by its expansion. However, a small clone can synthesize a very toxic protein, producing devastating systemic damage and protean clinical presentations. The resulting "monoclonal componentrelated diseases," although difficult to diagnose, may be progressive and even fatal. The monoclonal protein can aggregate and deposit systemically as occurs in light-chain amyloidosis, monoclonal immunoglobulin deposition disease, crystal-storing histiocytosis, and monoclonal cryoglobulinemia. Alternatively, some monoclonal proteins possess antibody activity toward autogenous antigens and cause chronic cold agglutinin disease, mixed cryoglobulinemia, and peripheral neuropathies. Other humoral mediators may contribute to neuropathy in variant disorders such as the POEMS (polyneuropathy, organomegaly, endocrinopathy, M protein, and skin changes) syndrome. The clone synthesizing the noxious monoclonal proteins is often small, and sensitive techniques may be required to detect these immunoglobulins. A delay in diagnosis can allow irreversible organ damage and dramatically shorten survival. Prompt recognition of suggestive signs and symptoms should trigger a thorough diagnostic approach to reach the correct diagnosis quickly, because this is the key to effective therapy. Although the treatment of these conditions is not optimal, significant advances have been made, improving the duration and quality of life.

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Section: Poems Syndromementioning

confidence: 99%

Dangerous small B-cell clones

Merlini

Stone

2006

Blood

394

280

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“…There are multiple cases describing some benefit using thalidomide, although there remains a legitimate concern for the development of disabling peripheral neuropathy (68)(69)(70)(71)(72). More recent data note benefit of autologous stem cell transplantation in recalcitrant cases (53,(73)(74)(75).…”

Section: Treatment and Prognosismentioning

confidence: 99%

Scleroderma-like Fibrosing Disorders

Boin¹,

Hummers²

2008

Rheumatic Disease Clinics of North America

109

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SynopsisMany conditions presenting with clinical hard skin and tissue fibrosis can be confused with systemic sclerosis (scleroderma). These disorders have very diverse etiologies and often an unclear pathogenetic mechanism. Distinct clinical characteristics, skin histology and disease associations may allow to distinguish these conditions from scleroderma and from each other. A prompt diagnosis is important to spare the patients from ineffective treatments and inadequate management. In this review, we highlighted nephrogenic systemic fibrosis (nephrogenic fibrosing dermopathy), eosinophilic fasciitis (Shulman's syndrome), scleromyxedema and scleredema. These are often detected in the primary care setting and referred to rheumatologists for further evaluation. Rheumatologists must be able to promptly recognize them to provide valuable prognostic information and appropriate treatment options for affected patients.

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“…Andere Behandlungsoptionen stellen die PUVA-Photochemotherapie sowie die systemische Gabe von Cyclosporin A, Retinoiden oder Antimalariamitteln dar. In Einzelfällen wurden auch Erfolge mit Thalidomid, der extrakorporalen Photophorese sowie der Gabe von Melphalan und begleitender autologer Stamzelltransplantation erzielt [1,3]. Bei einer Patientin mit ausgeprägter neurologischer Symptomatik erwiesen sich intravenös applizierte Immunglobuline effektiv [3].…”

Section: G Kukova · D Bruch-gerharz · K Gensch · T Ruzicka · J Runclassified