Erdheim-Chester-Erkrankung und unsere Erfahrungen mit Cladribin Therapie bei zwei Patienten

SZTURZ, Petr, Zdeněk ADAM, Renata KOUKALOVÁ, Zdeněk ŘEHÁK, Jiří NEUBAUER, Jiří PRÁŠEK, Luděk POUR, Lenka ZAHRADOVÁ, Sabina ŠEVČÍKOVÁ, Roman HÁJEK a Jiří MAYER. Erdheim-Chester-Erkrankung und unsere Erfahrungen mit Cladribin Therapie bei zwei Patienten. In Jahrestagung der Deutschen, Österreichischen und Schweizerischen Gesellschaften für Hämatologie und Onkologie. 2011.

Základní údaje

• Originální název: Erdheim-Chester-Erkrankung und unsere Erfahrungen mit Cladribin Therapie bei zwei Patienten
• Název anglicky: Erdheim-Chester disease and our experience with cladribine therapy in two patients
• Autoři: SZTURZ, Petr, Zdeněk ADAM, Renata KOUKALOVÁ, Zdeněk ŘEHÁK, Jiří NEUBAUER, Jiří PRÁŠEK, Luděk POUR, Lenka ZAHRADOVÁ, Sabina ŠEVČÍKOVÁ, Roman HÁJEK a Jiří MAYER.
• Vydání: Jahrestagung der Deutschen, Österreichischen und Schweizerischen Gesellschaften für Hämatologie und Onkologie, 2011.

Další údaje

• Typ výsledku: Konferenční abstrakt
• Utajení: není předmětem státního či obchodního tajemství
• Organizační jednotka: Lékařská fakulta
• UT WoS: 000295160600559

Anotace anglicky

Introduction: Erdheim-Chester disease is a rare idiopathic non-Langerhans cell histiocytosis with multiorgan involvement. It is characterized by proliferation of foamy histiocytes and their infiltration into various tissues and organs, typically the long bones of the lower extremities. Because of the low prevalence, there is no standard therapy of this disorder. Herein we report on the follow-up and treatment with cladribine (2-chloro-2-deoxyadenosine, 2-CdA, Litak)–based regimen in two patients with Erdheim-Chester disease and present an ample radiological documentation, including X-ray, CT, MRI, SPECT and PET/CT findings as well as those obtained by traditional bone scintigraphy. Materials and Methods: Two male patients, one born in 1953, the other in 1965, were diagnosed with Erdheim-Chester disease in 2008 and 2009, respectively. Both suffered from diabetes insipidus, fever or subfebrile temperatures, fatigue and pains in long bones of the lower extremities. They were treated with cladribine 5 mg/m2 SC, cyclophosphamide 150 mg/m2 IV (and dexamethasone 24 mg PO in the second patient) on days 1-5 of a 28-day cycle for 6 months in total. Bisphophonates (zoledronate 4 mg IV at 28-day intervals) as a long-term supportive therapy were administered. Results: In the first patient, remission of subjective symptoms as well as normalization of blood inflammatory markers and reduction of pathological hypermetabolism on restaging PET/CT examinations were achieved. In the second patient, bone pains regreded and restaging PET/CT showed stable disease. Conclusions: Treatment with cladribine–based regimens in two patients with Erdheim-Chester syndrome led to disease remission and stabilization, respectively. Therefore, we recommend cladribine to be standard part of treatments for this disease with potentionally life-threatening complications (pulmonary fibrosis, heart failure).