Rare Diseases Symptoms Automatic Extraction

Pentostatin, cyclophosphamide and rituximab is a safe and effective treatment in patients with Waldenström's macroglobulinemia.

[waldenström macroglobulinemia]

Waldenström's macroglobulinemia (WM) is an indolent lym phomaand is responsive to therapy regimens containing alkylating agents, purine analogs and rituximab if treatment becomes necessary. We initiated a multicenter phase II trial to determine the safety and efficacy of a regimen containing pentostatin, cyclophosphamide and rituximab (PER) in patients with WM. Between May 2005 and December 2010, 25 patients with WM were included in the study. Twenty-one patients received PER as first-line therapy. In these patients, 2-year progression-free survival was 83.6% and 2-year overall survival was 100%. Thirteen patients (52%) received R maintenance therapy. In these patients, the 2-year progression-free survival was 91.67% and 2-year overall survival was 100%. We have provided evidence that PER is a safe and effective regimen for WM. Although R maintenance therapy after PER seemed to induce a better long-term outcome, this study was not powered to address this issue.

Diseases presenting "long-term outcome" symptom

  • aniridia
  • cholangiocarcinoma
  • classical phenylketonuria
  • cystinuria
  • erythropoietic protoporphyria
  • esophageal squamous cell carcinoma
  • fabry disease
  • hirschsprung disease
  • homocystinuria without methylmalonic aciduria
  • hydrocephalus with stenosis of the aqueduct of sylvius
  • inclusion body myositis
  • kabuki syndrome
  • krabbe disease
  • oligodontia
  • papillon-lefèvre syndrome
  • primary hyperoxaluria type 1
  • trochlear dysplasia
  • von hippel-lindau disease
  • waldenström macroglobulinemia

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