RESUMO
A 40-year-old patient presented with rapidly progressing peripheral neuropathy secondary to monoclonal gammopathy of unknown significance (MGUS). He became severely debilitated, being wheelchair-bound, despite treatment with chemotherapy, intravenous immunoglobulin and plasma exchange. He was subsequently treated with high-dose chemotherapy followed by autologous peripheral blood stem cell transplantation (PBSCT). He has made remarkable recovery at 12 months post transplantation. We propose that high-dose chemotherapy and autologous PBSC transplantation may have a role in the treatment of severe, progressive and treatment-resistant MGUS-related peripheral neuropathy.
Assuntos
Gamopatia Monoclonal de Significância Indeterminada/complicações , Transplante de Células-Tronco de Sangue Periférico , Doenças do Sistema Nervoso Periférico/terapia , Adulto , Protocolos de Quimioterapia Combinada Antineoplásica/administração & dosagem , Humanos , Masculino , Condução Nervosa , Paraproteínas/metabolismo , Doenças do Sistema Nervoso Periférico/etiologia , Transplante Autólogo , Resultado do Tratamento , CaminhadaRESUMO
The safety, kinetics and efficacy of plerixafor+pegfilgrastim for hematopoietic stem and progenitor cell (HSPC) mobilization are poorly understood. We treated 12 study patients (SP; lymphoma n=10 or myeloma n=2) with pegfilgrastim (6 mg SC stat D1) and plerixafor (0.24 mg/kg SC nocte from D3). Six SP were 'predicted poor-mobilizers' and six were 'predicted adequate-mobilizers'. Peripheral blood (PB) CD34(+) monitoring commenced on D3. Apheresis commenced on D4. Comparison was with 22 historical controls (HC; lymphoma n=18, myeloma n=4; poor mobilizers n=4), mobilized with pegfilgrastim alone. Eight (67%) SP had PB CD34(+) count ⩽5 × 10(6)/L D3 post pegfilgrastim; all SP surpassed this threshold the morning after plerixafor. In SP, PBCD34(+) counts peaked D4 6/12 (50%), remaining ⩾5 × 10(6)/L for 4 days in 8/12 (67%). All SP successfully yielded target cell numbers (⩾2 × 10(6)/kg) within four aphereses. After maximum four aphereses, median total CD34+ yield was higher in SP than HC; 8.0 (range 2.4-12.9) vs 4.8 (0.4-14.0) × 10(6)/kg (P=0.04). Seven of twelve (58%) SP achieved target yield after one apheresis. Flow cytometry revealed no tumor cells in PB or apheresis product of SP. Plerixafor+pegfilgrastim was well tolerated with bone pain (n=2), diarrhoea (n=2) and facial paraesthesiae (n=3). Plerixafor+pegfilgrastim is a simple, safe and effective HSPC mobilization regimen in myeloma and lymphoma, in both poor and good mobilizers, and is superior to pegfilgrastim alone.
Assuntos
Fármacos Anti-HIV/administração & dosagem , Fator Estimulador de Colônias de Granulócitos/administração & dosagem , Mobilização de Células-Tronco Hematopoéticas/métodos , Compostos Heterocíclicos/administração & dosagem , Linfoma/terapia , Mieloma Múltiplo/terapia , Transplante de Células-Tronco de Sangue Periférico , Adulto , Autoenxertos , Benzilaminas , Remoção de Componentes Sanguíneos/métodos , Ciclamos , Feminino , Filgrastim , Humanos , Masculino , Pessoa de Meia-Idade , Polietilenoglicóis , Proteínas Recombinantes/administração & dosagemRESUMO
Fludarabine combination chemotherapy achieves high response rates in chronic lymphocytic leukemia (CLL) and indolent lymphoma. The aim of this study was to investigate the incidence and characteristics of treatment-related myelodysplasia and acute myeloid leukemia (t-MDS/AML) after treatment with fludarabine in combination for lymphoproliferative disorders and identify risk factors for its development. In all, 176 patients treated with fludarabine combination were followed for a median of 41 months (range 6-125 months). In all, 19 cases of t-MDS/AML have been identified for an overall rate of 10.8%. Median overall survival post-t-MDS/AML diagnosis was 11 months. Patients developing t-MDS/AML included 11/54 with follicular lymphoma (FL) (crude rate 20.4%), 5/82 with CLL (6.1%) and 3/24 with Waldenstrom macroglobulinemia or marginal zone lymphoma (12.5%). Most patients had other cytotoxic treatments (median 4, range 0-7) but three with FL had fludarabine combination as their only line of treatment. Of the eleven patients (6.3%) who received mitoxantrone with their first fludarabine combination, four (36.4%) developed t-MDS/AML (P=0.007). There was a trend toward prior cytotoxic therapy increasing the risk for t-MDS/AML (P=0.067). Fludarabine combination chemotherapy is associated with a moderate risk of t-MDS/AML particularly when combined with mitoxantrone. This complication should be considered when evaluating the potential benefit of this treatment in lymphoproliferative disorders.
Assuntos
Antineoplásicos/efeitos adversos , Leucemia Mieloide Aguda/induzido quimicamente , Síndromes Mielodisplásicas/induzido quimicamente , Segunda Neoplasia Primária/induzido quimicamente , Vidarabina/análogos & derivados , Adulto , Idoso , Idoso de 80 Anos ou mais , Feminino , Transplante de Células-Tronco Hematopoéticas , Humanos , Leucemia Mieloide Aguda/mortalidade , Leucemia Mieloide Aguda/terapia , Masculino , Pessoa de Meia-Idade , Síndromes Mielodisplásicas/mortalidade , Síndromes Mielodisplásicas/terapia , Vidarabina/efeitos adversosRESUMO
MonoFIX-VF, a monocomponent factor IX concentrate, has replaced the use of Prothrombinex-HT as the treatment of choice for patients with factor IX deficiency in Australia. The haemostatic effect of MonoFIX-VF, administered by continuous infusion, was assessed in four subjects being treated for 10 bleeding episodes including five surgical procedures. MonoFIX-VF was found to be a safe and effective treatment for patients with haemophilia B.