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Syndroom van POEMS : Verslag van een zeldzame plasmacel dyscrasie in Nederland

(2016) Langbroek, P.

Summary
POEMS syndrome is a rare plasma cell dyscrasia characterized by polyneuropathy, sclerotic bone lesions, Castleman disease, elevated VEGF levels, organomegaly, extravascular volume overload, endocrinopathy, skin changes, papilledema, thrombocytosis and polycythemia. In this retrospective study an overview is presented of the characteristics, treatment and course of patients diagnosed with POEMS syndrome in the Netherlands. 27 patients were included. The mean age was 51 years, 67% was male. The median time between onset of disease and diagnosis was 15 months (range 2-36 months). 74% presented with polyneuropathy as one of the initial symptoms. The most frequent characteristics besides a plasma cell dyscrasia and polyneuropathy (mandatory for diagnosis) were elevated VEGF levels (100%), skin changes (78%), endocrinopathy (71%) and sclerotic bone lesions (70%). The median follow-up time was 32 months. Lenalidomide/Dexamethasone therapy was used in 56% of patiënts and was effective in 73%. High-dose melphalan followed by an autologous stem cell re-infusion was the most frequent (65%) and most effective (100%) treatment. Eventually 65% of patient had a hematologic response, 100% a VEGF-response and 71% a neurologic response, while 88% of treatments were successfully completed. 1 patient died as a consequence of the disease. Treatment with corticosteroids alone appeared to be the only treatment with a negative effect on the hematologic response (p=0,04). The time to diagnosis, disease-characteristics and the other treatments were not associated with the clinical, haematologic, neurologic or VEGF-response. In conclusion, when diagnosis is finally made, treatment can be very effective. The proposed responses for disease follow-up seem to be lacking an association with therapy effectiveness.





ID 3405
Moeder ID 3084
Volgorde langbroek, P.
Naam LangbroekP
Publiceren yes
OAI-naam Student_thesis
Path root/geneeskunde/2016/LangbroekP/
Gemaakt op: 2017-05-30 11:59:43
Gemodificeerd op: 2017-05-30 11:59:43
Digitaal ID 592d5eb5b2ab0
Afstudeerrichting opleiding/afstudeerrichting 1
Studierichting Studierichting 1
Titel Syndroom van POEMS : Verslag van een zeldzame plasmacel dyscrasie in Nederland
Ruilverkeer mogelijk no
Printen in opdracht no
Aantal pagina's 39
Publicatiejaar 2016
Taal nl
Engelse samenvatting Summary
POEMS syndrome is a rare plasma cell dyscrasia characterized by polyneuropathy, sclerotic bone lesions, Castleman disease, elevated VEGF levels, organomegaly, extravascular volume overload, endocrinopathy, skin changes, papilledema, thrombocytosis and polycythemia. In this retrospective study an overview is presented of the characteristics, treatment and course of patients diagnosed with POEMS syndrome in the Netherlands. 27 patients were included. The mean age was 51 years, 67% was male. The median time between onset of disease and diagnosis was 15 months (range 2-36 months). 74% presented with polyneuropathy as one of the initial symptoms. The most frequent characteristics besides a plasma cell dyscrasia and polyneuropathy (mandatory for diagnosis) were elevated VEGF levels (100%), skin changes (78%), endocrinopathy (71%) and sclerotic bone lesions (70%). The median follow-up time was 32 months. Lenalidomide/Dexamethasone therapy was used in 56% of patiënts and was effective in 73%. High-dose melphalan followed by an autologous stem cell re-infusion was the most frequent (65%) and most effective (100%) treatment. Eventually 65% of patient had a hematologic response, 100% a VEGF-response and 71% a neurologic response, while 88% of treatments were successfully completed. 1 patient died as a consequence of the disease. Treatment with corticosteroids alone appeared to be the only treatment with a negative effect on the hematologic response (p=0,04). The time to diagnosis, disease-characteristics and the other treatments were not associated with the clinical, haematologic, neurologic or VEGF-response. In conclusion, when diagnosis is finally made, treatment can be very effective. The proposed responses for disease follow-up seem to be lacking an association with therapy effectiveness.
Nederlandse samenvatting Het syndroom van POEMS is een zeldzame plasmaceldyscrasie gekenmerkt door polyneuropathie, sclerotische bothaarden, de ziekte van Castleman, verhoogde VEGF concentratie, organomegalie, overvullingskenmerken, endocrinopathie, huidveranderingen, papiloedeem, trombocytose en polycythemie. Deze retrospectieve studie geeft een overzicht van de kenmerken, de behandeling en het ziektebeloop van patiënten met het syndroom van POEMS in Nederland. 27 patiënten werden geïncludeerd. De gemiddelde leeftijd was 51 jaar, 67% was man. De mediane tijd tussen het ontstaan van klachten en de diagnose was 15 maanden (range 2-36 maanden). 74% presenteerde zich met polyneuropathie als één van de eerste klachten. Veelvoorkomende kenmerken naast plasmaceldyscrasie en polyneuropathie (verplicht voor diagnose) waren een verhoogd VEGF (100%), huidafwijkingen (78%), endocrinopathie (71%) en sclerotische bothaarden (70%). De mediane follow-up tijd was 32 maanden. Lenalidomide/Dexamethasontherapie werd gegeven aan 56% van de patiënten en was effectief bij 73%. Hoge dosering melfalan gevolgd door een autologe stamcel reinfusie was de meest frequent toegepaste (65%) en meest effectieve (100%) behandeling. Uiteindelijk had 65% van de patiënten enige hematologische respons, 100% enige VEGF-respons en 71% enige neurologische respons, terwijl 88% van de patiënten de behandelingen met goede effectiviteit had afgerond. 1 patiënt overleed ten gevolge van de ziekte. Behandeling met corticosteroïden alleen werd als enige geassocieerd met een slechtere hematologische respons (p=0,04). De tijd tot diagnose, ziekte-kenmerken en de andere behandelingen hielden geen verband met de klinische, hematologische, neurologische en VEGF-respons. Concluderend kan worden gesteld dat, wanneer de diagnose eenmaal gesteld is, behandeling zeer effectief is. De eerder voorgestelde responsen voor het vervolgen van de ziekte lijken geen goede weerslag te geven van de effectiviteit van de behandeling.
Onderwijsinstelling Medical Sciences
Type embargo abstract openbaar, scriptie op aanvraag
Auteur(s) Langbroek, P.
UMCG begeleider(s) Facultair begeleider:; Waal, Mw. drs. E.G.M. deinternist-hematoloog MCL; Tweede begeleider:; Vellenga, Prof. dr. E. hematoloog UMCG
Auteur(s) Langbroek, P.
UMCG begeleider(s) Facultair begeleider:; Waal, Mw. drs. E.G.M. deinternist-hematoloog MCL; Tweede begeleider:; Vellenga, Prof. dr. E. hematoloog UMCG


 
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