POEMS syndrome treated with melphalan high-dose therapy and autologous blood stem cell transplantation: a single-institution experience
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POEMS syndrome treated with melphalan high-dose therapy and autologous blood stem cell transplantation: a single-institution experience. / Thoennissen, Gabriela B; Thoennissen, Nils H; Fritz, Fleur; Hilbig, Andreas; Kerkhoff, Andrea; Liersch, Ruediger; Krug, Utz; Koschmieder, Steffen; Müller-Tidow, Carsten; Mesters, Rolf; Kropff, Martin; Berdel, Wolfgang E.
in: ANN HEMATOL, Jahrgang 91, Nr. 9, 09.2012, S. 1419-25.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
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TY - JOUR
T1 - POEMS syndrome treated with melphalan high-dose therapy and autologous blood stem cell transplantation: a single-institution experience
AU - Thoennissen, Gabriela B
AU - Thoennissen, Nils H
AU - Fritz, Fleur
AU - Hilbig, Andreas
AU - Kerkhoff, Andrea
AU - Liersch, Ruediger
AU - Krug, Utz
AU - Koschmieder, Steffen
AU - Müller-Tidow, Carsten
AU - Mesters, Rolf
AU - Kropff, Martin
AU - Berdel, Wolfgang E
PY - 2012/9
Y1 - 2012/9
N2 - The acronym POEMS syndrome stands for a rare multi-system disorder, comprised of polyneuropathy, organomegaly, endocrinopathy, M protein, and skin changes. Here, we present a single-center report of a series of five POEMS patients treated with melphalan high-dose therapy (HDT) with subsequent autologous blood stem cell transplantation (ABSCT). After a median follow-up of 52 months from time of diagnosis (range, 15-192) and a median follow-up of 18 months after ABSCT (range, 11-120), all patients were alive. Overall, no severe transplantation-associated complications such as engraftment syndrome or peri- or post-transplant death were noted. In two cases, HDT followed by ABSCT resulted in a complete hematologic response; in the additional three cases, partial responses (PR) were achieved including one very good hematologic PR. Only one patient with initial PR developed progressive disease nearly 2.5 years after transplantation. Consequently, a second HDT with ABSCT was successfully applied resulting in clinical improvement and hematologic PR. In line with previous single-center reports, melphalan HDT followed by ABSCT proved to be a first-line treatment option with tolerable side effects in severely affected POEMS patients with progressing symptoms.
AB - The acronym POEMS syndrome stands for a rare multi-system disorder, comprised of polyneuropathy, organomegaly, endocrinopathy, M protein, and skin changes. Here, we present a single-center report of a series of five POEMS patients treated with melphalan high-dose therapy (HDT) with subsequent autologous blood stem cell transplantation (ABSCT). After a median follow-up of 52 months from time of diagnosis (range, 15-192) and a median follow-up of 18 months after ABSCT (range, 11-120), all patients were alive. Overall, no severe transplantation-associated complications such as engraftment syndrome or peri- or post-transplant death were noted. In two cases, HDT followed by ABSCT resulted in a complete hematologic response; in the additional three cases, partial responses (PR) were achieved including one very good hematologic PR. Only one patient with initial PR developed progressive disease nearly 2.5 years after transplantation. Consequently, a second HDT with ABSCT was successfully applied resulting in clinical improvement and hematologic PR. In line with previous single-center reports, melphalan HDT followed by ABSCT proved to be a first-line treatment option with tolerable side effects in severely affected POEMS patients with progressing symptoms.
KW - Adult
KW - Anthracyclines
KW - Combined Modality Therapy
KW - Dexamethasone
KW - Female
KW - Humans
KW - Male
KW - Melphalan
KW - Middle Aged
KW - Myeloablative Agonists
KW - Neurologic Examination
KW - POEMS Syndrome
KW - Peripheral Blood Stem Cell Transplantation
KW - Transplantation, Autologous
KW - Treatment Outcome
KW - Vascular Endothelial Growth Factor A
U2 - 10.1007/s00277-012-1473-7
DO - 10.1007/s00277-012-1473-7
M3 - SCORING: Journal article
C2 - 22543828
VL - 91
SP - 1419
EP - 1425
JO - ANN HEMATOL
JF - ANN HEMATOL
SN - 0939-5555
IS - 9
ER -