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ABSTRACT: Antifungal prophylaxis in the haematological patient is currently regarded as the gold standard in situations with a high risk of infection, such as acute leukaemias, myelodysplastic syndromes and autologous or allogenic hematopoietic stem cell transplantation. Over the years, different scientific societies have established a series of recommendations on antifungal prophylaxis based on prospective studies performed with different drugs. However, the prescription of each one of the agents must be personalised, adapted to the characteristics of each patient and to possible interactions with concomitant medication.
Revista espanola de quimioterapia: publicacion oficial de la Sociedad Espanola de Quimioterapia 12/2012; 25(4):299-304. · 0.81 Impact Factor
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José Barberán,
Josep Mensa,
Juan Carlos Vallejo Llamas, Isidro Jarque Ramos,
Juan Carlos García Ruiz,
José Rafael Cabrera Marín,
Patricia Baltasar Tello,
Montse Batlle Massana,
Juan Besalduch Vidal,
José Manuel Calvo Viñas, [......],
Ramón Cisterna Cáncer,
Jesús Fortún Abete,
Julio García Rodríguez,
Joaquín Gómez Gómez,
Elia Gómez García de la Pedrosa,
José Miguel Montejo Baranda,
F Javier Pemán García,
Isabel Ruiz Camps,
Miguel Salavert Lleti,
Julián de la Torre Cisneros
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ABSTRACT: Antifungal treatment in the hematological patient has reached a high complexity with the advent of new antifungals and diagnostic tests, which have resulted in different therapeutic strategies. The use of the most appropriate treatment in each case is essential in infections with such a high mortality. The availability of recommendations as those here reported based on the best evidence and developed by a large panel of 48 specialists aimed to answer when is indicated to treat and which agents should be used, considering different aspects of the patient (risk of fungal infection, clinical manifestations, galactomanann test, chest CT scan and previous prophylaxis) may help clinicians to improve the results.
Revista espanola de quimioterapia: publicacion oficial de la Sociedad Espanola de Quimioterapia 12/2011; 24(4):263-70. · 0.81 Impact Factor
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ABSTRACT: Posaconazole has been proven to be as effective as fluconazole in the prevention of invasive fungal infections (IFI) in allogeneic haematopoietic SCT patients with GVHD. We assessed, from the perspective of the Spanish National Health Service, the cost-effectiveness of posaconazole vs fluconazole in preventing IFI. A decision-analytic model was developed to assess the average per patient treatment costs, IFIs avoided, life-years gained (LYG) and incremental cost per LYG for each prophylactic treatment used (in euros at 2007 prices). Patients are assumed to have received either posaconazole or fluconazole. The probabilities of IFI, IFI-related death and death from other causes were obtained from a single clinical trial. Long-term mortality and costs were estimated from secondary sources. Posaconazole was associated with fewer IFIs (5.3 vs 9%), increased LYG (8.01 vs 7.78) and higher IFI-related costs ([euro]11 585 vs [euro]6 959) per patient compared with fluconazole. The incremental cost-effectiveness of posaconazole vs fluconazole was estimated at [euro]20 246 per LYG. There was a 70% probability that posaconazole is cost-effective at a [euro]30 000 per LYG threshold. In conclusion, compared with fluconazole, posaconazole prophylaxis is a cost-effective strategy for the prevention of IFI in patients with GVHD.
Bone marrow transplantation 10/2009; 45(5):925-32. · 3.00 Impact Factor
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ABSTRACT: Meningoencephalitis caused by pathogenic free-living amebas is usually fatal. Only a few cases of Acanthamoeba meningoencephalitis, diagnosed at autopsy, have been reported following hematopoietic stem cell transplantation. We here report a case of Acanthamoeba meningoencephalitis following allogeneic peripheral blood stem cell transplantation with rapidly evolving neurologic symptoms that remained unexplained. Magnetic resonance imaging failed to show brain lesions and cerebrospinal fluid was negative for microbiological cultures. Definite diagnosis was an unexpected autopsy finding. As overall and teaching hospital autopsy rates are declining worldwide, we must emphasize the need of autopsy exams if we want to improve our knowledge as the best way to care for our patients.
American Journal of Transplantation 06/2008; 8(7):1562-6. · 6.39 Impact Factor
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A Moreno,
C Cervera,
J Gavaldá,
M Rovira,
R de la Cámara, I Jarque,
M Montejo,
J de la Torre-Cisneros,
J Miguel Cisneros,
J Fortún,
F López-Medrano,
M Gurguí,
P Muñoz,
A Ramos,
J Carratalá
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ABSTRACT: Bloodstream infections (BSIs) are a major cause of morbidity and mortality in transplant recipients. The aim of this study is to describe the incidence, microbiology and outcomes of BSIs in transplant recipients in Spain. The Spanish Network for Research on Infection in Transplantation (RESITRA) is formed by 16 centers with transplant program in Spain. The incidence and characteristics of BSIs in transplant patients were obtained prospectively from the cohort. We included 3926 transplant recipients (2935 solid organ and 991 hematopoietic stem cell transplants). Overall, 730 episodes of BSIs were recorded with an incidence rate ranging from 3 episodes per 10 000 transplant days in kidney recipients to 44 episodes per 10 000 transplant days in allogeneic hematopoietic stem cell transplantation (HSCT). The most frequent sources were intravascular catheters and the most frequent microorganisms isolated were coagulase-negative staphylococci. Crude mortality of BSIs was 7.8%, being highest in liver recipients (16%). Multidrug resistant nonfermentative gram-negative BSIs had significantly worse prognosis than those caused by their susceptible counterparts (p = 0.015), but no differences were found between resistant and susceptible gram-negative enteric bacilli, S. aureus or Candida spp. BSIs are still a major concern in transplant recipients. The increasing isolations of multiresistant microorganisms represent a challenge for the next years.
American Journal of Transplantation 12/2007; 7(11):2579-86. · 6.39 Impact Factor
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ABSTRACT: In order to take the best approach to infection in the oncohematologic patient with fever, it is important to know not only how profound the neutropenia is and how long the patient has had it, but also the characteristics of the underlying disease, the immunosuppressive therapy received and the type of hematopoietic stem/progenitor cell transplantation performed. Moreover, is important to consider if these patients have any personal or familial history of infectious diseases. All these aspects let us calculate the net state of immunosuppression and the risk of infection, and provide us with information about the most probable etiology in each case and the best prophylaxis and treatment. In this study we review the more important advances in chemotherapy in recent years that will make it necessary in the future to change our prophylactic guidelines for more effective prevention of infection in the oncohematologic patient.
Revista espanola de quimioterapia: publicacion oficial de la Sociedad Espanola de Quimioterapia 07/2006; 19(2):117-29. · 0.81 Impact Factor
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Revista espanola de quimioterapia: publicacion oficial de la Sociedad Espanola de Quimioterapia 04/2006; 19(1):11-3. · 0.81 Impact Factor
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J de la Rubia,
S Cantero,
G F Sanz,
M J Remigia,
E Monteagudo,
F Moscardó,
G Martín,
I Lorenzo,
C Jiménez,
J Martínez,
P Montesinos, I Jarque,
M A Sanz
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ABSTRACT: We evaluated the use of CD34+ selected allogeneic peripheral blood as a source of hematopoietic progenitors for allogeneic transplantation in 11 patients with aplastic anemia (AA). The median age was 17 years (range, 6--9), and the median time between diagnosis and transplant 1 month (range, 1--4). Conditioning consisted of cyclophosphamide (50 mg/kg per day) on days--7 to--4 and antithymocyte globulin (30 mg/kg per day) on days--4 to--2 in nine patients. Total lymphoid irradiation was added to the preparative regimen for two. Graft-versus-host disease (GVHD) prophylaxis consisted of cyclosporine A and prednisone. Median doses of CD34+ and CD3+ cells infused were 3.91 x 10(6) and 0.3 x 10(6)/kg, respectively. The median time taken to achieve a neutrophil count >0.5 x 10(9)/l was 12 days and to recover a platelet count >20 x 10(9)/l, 13 days. Two patients developed acute GVHD grade I--II and one developed limited chronic GVHD. There were two treatment-related deaths. At a median follow-up of 44 months (range, 4--3), nine patients were alive with sustained and complete engraftment. This is a promising procedure in patients with AA, resulting in a rapid hematopoietic recovery, a low transplant-related mortality, and a low incidence of GVHD.
Bone Marrow Transplantation 09/2005; 36(4):325-9. · 3.75 Impact Factor
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A Sureda,
M Constans,
A Iriondo,
R Arranz,
M D Caballero,
M J Vidal,
J Petit,
A López,
J J Lahuerta,
E Carreras, [......],
C Albó,
J Díaz-Mediavilla,
A Torres,
P Torres,
J Besalduch,
J Marín,
M V Mateos,
J M Fernández-Rañada,
J Sierra,
E Conde
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ABSTRACT: To analyse outcome and prognostic factors for overall survival (OS) and time to treatment failure (TTF) in 357 patients with Hodgkin's lymphoma (HL) undergoing an autologous stem cell transplantation (ASCT) after a first relapse and reported to the The Grupo Espanol de Linfomas/Trasplante Autologo de Medula Osea (GEL/TAMO) Cooperative Group.
Two hundred and twenty males and 137 females with a median age of 29 years were autografted in second remission (n=181), first sensitive relapse (n=148) and first resistant relapse (n=28).
Five-year actuarial TTF and OS were of 49% +/- 3% and 57% +/- 3%. Advanced stage at diagnosis, complementary radiotherapy before ASCT, a short first complete response (CR) and detectable disease at ASCT adversely influenced TTF. Year of transplant < or =1995, bulky disease at diagnosis, a short first CR, detectable disease at ASCT and > or =1 extranodal areas involved at ASCT were adverse factors for OS.
ASCT constitutes a therapeutic option for HL patients after a first relapse. Promising results are observed in patients with low tumour burden at diagnosis, autografted after a long CR and without detectable disease at ASCT. Innovative approaches should be pursued for patients with risk factors at relapse.
Annals of Oncology 05/2005; 16(4):625-33. · 6.43 Impact Factor
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I Jarque,
M Salavert,
E Romá,
M Gobernado,
M A Chiveli,
M Ruano,
A Solé,
J L Poveda,
E Ferrer,
J García-Pellicer,
M A Sanz,
J Pemán,
V Sánchez
Revista espanola de quimioterapia: publicacion oficial de la Sociedad Espanola de Quimioterapia 01/2005; 17(4):357-89. · 0.81 Impact Factor
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J Rodriguez,
M D Caballero,
A Gutierrez,
C Solano,
R Arranz,
J J Lahuerta,
J Sierra,
M Gandarillas,
J A Perez-Simon,
J Zuazu, [......],
J Marin,
J C Garcia,
A Fernandez De Sevilla,
J Rifon,
R Varela, I Jarque,
C Albo,
A Leon,
J SanMiguel,
E Conde
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ABSTRACT: Here we evaluate the results of high-dose chemotherapy and autologous stem-cell transplantation (HDC/ASCT) in 114 patients included in the GEL/TAMO registry between January 1990 and December 1999 with diffuse large B-cell lymphoma who failed to achieve complete remission (CR) with front-line conventional chemotherapy.
Sixty-eight per cent had a partial response (PR) and 32% failed to respond to front-line therapy. At transplant, 35% were chemoresistant and 29% had two to three adjusted International Prognostic Index (a-IPI) risk factors.
After HDC/ASCT, 57 (54%) of 105 patients evaluable for response achieved a CR, 16 (15%) a PR and 32 (30%) failed. Nine patients were not assessed for response because of early death due to toxicity. With a median follow-up of 29 months for alive patients, the survival at 5 years is 43%, with a disease-free survival for complete responders of 63%. The lethal toxicity was 8%. Multivariate analysis revealed a-IPI and chemoresistance to be predicting factors.
Our results show that one-third of patients who do not obtain a CR to front-line chemotherapy may be cured of their disease with HDC/ASCT. However, most chemoresistant patients pretransplant failed this therapy. For this population, as well as for those who presented with adverse factors of the a-IPI, pretransplant novel therapeutic modalities need to be tested.
Annals of Oncology 11/2004; 15(10):1504-9. · 6.43 Impact Factor
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J de la Rubia,
G Martín,
J Martínez,
I Lorenzo,
G Sanz, I Jarque,
F Moscardó,
C Jiménez,
P Lorente,
A Camps,
M A Sanz
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ABSTRACT: Different strategies for collecting peripheral blood stem cells (PBSC) for autologous blood stem cell transplantation (ABSCT) have been reported for patients with acute myeloblastic leukemia (AML). We compared the clinical results of 2 consecutive protocols in 75 adult patients with AML in first complete remission who underwent ABSCT. In the first 56 patients (group A), PBSC were collected after induction and/or consolidation chemotherapy courses. In the subsequent 19 patients (group B), PBSC collection was done after a further intensification course with intermediate-dose cytarabine and mitoxantrone. Hematopoietic engraftment was similar in the 2 groups, with the median times to reach 0.5 x 10(9) neutrophils/L and 20 x 10(9) platelets/L being 13 days each in group A, and 12 days and 24 days, respectively, in group B. There were 3 graft failures (all in group A) and 5 transplantation-related deaths (6.6%, 4 in group A and 1 in group B). Although not statistically significant, the 3-year probabilities of both relapse (31% versus 66%; P = .12) and disease-free survival (60% versus 36%; P = .1) compared favorably for group B. Our study suggests that collection of PBSC after additional intensification can result in a better outcome for AML patients who undergo ABSCT.
International Journal of Hematology 09/2004; 80(2):168-73. · 1.27 Impact Factor
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I Jarque
Revista espanola de quimioterapia: publicacion oficial de la Sociedad Espanola de Quimioterapia 04/2004; 17(1):94-7. · 0.81 Impact Factor
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M Constans,
A Sureda,
M J Terol,
R Arranz,
M D Caballero,
A Iriondo, I Jarque,
E Carreras,
J M Moraleda,
D Carrera, [......],
F Hernández-Navarro,
R Mataix,
J Petit,
M J Pascual,
J Rifón,
J García-Conde,
J M Fernández-Rañada,
M V Mateos,
J Sierra,
E Conde
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ABSTRACT: Patients with primary refractory Hodgkin's disease (PR-HD) have a dismal prognosis when treated with conventional salvage chemotherapy. We analyzed time to treatment failure (TTF), overall survival (OS) and clinical variables influencing the outcome in patients undergoing autologous stem cell transplantation (ASCT) for PR-HD and reported to the Grupo Español de Linfomas/Trasplante Autólogo de Médula Osea (GEL/TAMO).
Sixty-two patients, 41 males and 21 females with a median age of 27 years (range 13-55) were analyzed. Forty-two patients (68%) had advanced stage at diagnosis, 47 (76%) presented with B symptoms and 29 (47%) with a bulky mediastinal mass. Seventy-five percent of the patients had received more than one line of therapy before ASCT. Thirty-three patients received bone marrow as a source of hematopoietic progenitors, and 29 peripheral blood. Six patients were conditioned with high-dose chemotherapy plus total-body irradiation and 56 received chemotherapy-based protocols.
One-year transplantation-related mortality was 14% [95% confidence interval (CI) 6% to 23%]. Response rate at 3 months after ASCT was 52% [complete remission in 21 patients (34%), partial remission in 11 patients (18%)]. Actuarial 5-year TTF and OS were 15% (95% CI 5% to 24%) and 26% (95% CI 13% to 39%), respectively. The presence of B symptoms at ASCT was the only adverse prognostic factor significantly influencing TTF [relative risk (RR) 1.75, 95% CI 0.92-3.35, P = 0.08]. The presence of B symptoms at diagnosis (RR 2.08, 95% CI 0.90-4.79, P = 0.08), MOPP-like regimens as first-line therapy (RR 3.84, 95% CI 1.69-9.09, P = 0.001), bulky disease at ASCT (RR 2.79, 95% CI 0.29-6.03, P = 0.009) and two or more lines of therapy before ASCT (RR 2.24, 95% CI 0.95-5.27, P = 0.06) adversely influenced OS.
In our experience, although overall results of ASCT in PR-HD patients are poor, one-quarter of the patients remain alive at 5 years. Despite this, other therapeutic strategies should be investigated in this group of patients to improve the outcome.
Annals of Oncology 06/2003; 14(5):745-51. · 6.43 Impact Factor
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S Saavedra,
G F Sanz, I Jarque,
F Moscardó,
C Jiménez,
I Lorenzo,
G Martín,
J Martínez,
J De La Rubia,
R Andreu,
S Mollá,
I Llopis,
M J Fernández,
M Salavert,
B Acosta,
M Gobernado,
M A Sanz
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ABSTRACT: Early transplant-related mortality after cord blood transplantation from unrelated donors (UD-CBT) is close to 50%, mainly due to infectious complications. We have studied the incidence and characteristics of early infections (before day 100) in a series of 27 adult patients (median age 30 years, range 16-46) undergoing UD-CBT at a single institution. All 27 patients experienced at least one infectious episode and 18 (66%) suffered a severe infection. Bacteremia occurred in 55% of patients (13 with Gram-positive and 11 with Gram-negative microorganisms). Eleven of 19 CMV-seropositive patients (58%) developed CMV antigenemia and one patient had CMV disease. Fungal infections were documented in three patients (11%), comprising invasive fungal infections in two cases and a localized esophagitis in one. Ten patients (37%) died before day 100 after transplantation. Infection was considered the primary cause of death in four patients (sepsis by Acinetobacter spp. bacteremia in three cases) and contributed to death in another four. The most striking findings in this series were the high incidence of, and mortality due to multiresistant Acinetobacter spp. and the low incidence of and lack of mortality due to CMV disease. This report confirms that infection is a major complication in adults undergoing UD-CBT.
Bone Marrow Transplantation 01/2003; 30(12):937-43. · 3.75 Impact Factor
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ABSTRACT: Factors influencing the collection of autologous peripheral blood stem cells (PBSCs) were studied in 182 mobilization procedures performed on 145 consecutive patients with acute myeloblastic leukemia (AML; n=67) and with various non-myeloid malignancies (NMM; n=78). PBSC were collected following mobilization with chemotherapy, treatment with granulocyte colony-stimulating factor (G-CSF) or chemotherapy plus G-CSF. Fewer colony-forming unit granulocyte-macrophages (CFU-GMs) were collected from patients with AML than from patients with NMM (P<0.0001), although there were no differences in the numbers of CD34+ cells collected between both groups. Multiple regression analysis showed that chemotherapy alone was predictive of a low CD34+ yield in patients with NMM (regression coefficient (RC)=-2.1; P=0.003). In addition, the interactions "diagnosis mutliple myeloma (MM)xmobilization with chemotherapy" (RC=2.9; P=0.004) and "diagnosis MMxmobilization with chemotherapy plus G-CSF" (RC=2.1; P=0.04) also remained in the model, both showing a favorable influence. In AML, mobilization with chemotherapy plus G-CSF was associated with higher CD34+ yields (P=0.003). In this subgroup of patients, multiple regression analysis identified the number of cycles of previous chemotherapy (< or =2 cycles; RC=1.3; P=0.03) and peripheral blood counts (WBC > or =1.5 x 10(9)/l and monocytes >20%; RC=0.8; P=0.02) as the factors most predictive of CD34+ cell yield. These findings emphasize the need to optimize harvesting technique to enhance safety and minimize morbidity and costs of this valuable procedure.
Leukemia Research 01/2003; 27(1):5-12. · 2.92 Impact Factor
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S Saavedra, I Jarque,
G F Sanz,
F Moscardó,
C Jiménez,
G Martín,
G Plumé,
A Regadera,
J Martínez,
J De La Rubia,
B Acosta,
J Pemán,
C Pérez-Bellés,
M Gobernado,
M A Sanz
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ABSTRACT: To analyze the incidence and characteristics of documented infections in patients with hematologic malignancies undergoing unrelated donor bone marrow transplantation (UD-BMT).
We studied the occurrence of infections in 22 patients with hematologic malignancies or severe aplastic anemia who underwent UD-BMT from April 1990 to December 2000. The median age was 26 years (range 13-46). Acyclovir-ganciclovir, co-trimoxazole, fluconazole-nystatin and ciprofloxacin were administered for anti-infectious prophylaxis.
We registered 61 infectious episodes. During the early post-transplant period, there were eight clinically documented infections (CDIs), four cases of fever of unknown origin (FUO), seven cases of bacteremia, two cases of cytomegalovirus (CMV) antigenemia, and one case of CMV disease. In the intermediate period (days 30-100 after BMT), there were nine cases of CMV antigenemia, three bacterial infections, two fungal infections, one case of disseminated toxoplasmosis, and one case of FUO. In the late period (day 100 and later), we documented 13 viral infections, eight bacterial infections, one CDI, and one case of invasive aspergillosis. Infections contributed to death in 10 of 17 patients. Citrobacter bacteremia and sepsis of unknown origin were the main causes of infectious mortality in the early period. Infection was the main cause of death in six of seven patients in the late period.
A high incidence of life-threatening infections and infection-related mortality was observed. A high rate of CMV infection in the early period, and death caused by multiresistant Gram-negative microorganisms in the late period, were the main findings in this series.
Clinical Microbiology and Infection 12/2002; 8(11):725-33. · 4.54 Impact Factor
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ABSTRACT: Factors influencing hematopoietic recovery (HR) after autologous blood stem cell transplantation (ABSCT) were analyzed in 73 patients with various non-myeloid malignancies (NMM), and in 58 patients with acute myeloblastic leukemia (AML). Peripheral blood stem cells were collected following mobilization with chemotherapy, granulocyte colony-stimulating factor (G-CSF), or chemotherapy plus G-CSF. The conditioning regimen used consisted of either chemotherapy alone (112 cases) or chemotherapy plus total body irradiation (19 cases). The median number of colony-forming units granulocyte-macrophage (CFU-GM) was similar in both groups of patients, with the median number of CD34(+) cells infused being higher in the AML group (5.4 vs 4 x 10(6)/kg; P = 0.03). Median time neutrophils >0.5 x 10(9)/l was 13 days in both groups, and median time to a platelet count >20 x 10(9)/l was longer in AML patients (14 vs 12 days; P = 0.01). In multivariate analysis, the only factors affecting neutrophil recovery in the NMM group were the CD34+ cell number (continuous model) and the CFU-GM dose (categorized model) infused, whereas for platelet recovery, previous chemotherapy also remained significant. In the AML group, the only factors significantly affecting the speed of neutrophil recovery were dose of CD34+ cells administered and the patient's age. As for platelet recovery, only the progenitor dose administered remained significant. In the NMM group, the most discriminating cut-off values for a rapid neutrophil and platelet recovery were 1.5 x 10(6) and 2.5 x 10(6) CD34+ cells/kg, respectively, and for AML patients these figures were 1.5 x 10(6) and 4 x 10(6) CD34+ cells/kg, respectively. Our results confirm the slower HR after ABSCT in AML, and highlight the importance of progenitor cell dose in accelerating HR after ABSCT.
Bone Marrow Transplantation 06/2002; 29(10):825-32. · 3.75 Impact Factor
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I Jarque,
R Andreu,
I Llopis,
J De la Rubia,
F Gomis,
L Senent,
C Jiménez,
G Martín,
J A Martínez,
G F Sanz,
J Ponce,
M A Sanz
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ABSTRACT: Eradication of Helicobacter pylori infection has been associated with the correction of thrombocytopenia in patients with idiopathic thrombocytopenic purpura (ITP). We have analysed the response to eradication of H. pylori in a series of 56 adult patients with chronic ITP. Forty patients had H. pylori infection (71%) that was eradicated in 23 of 32 evaluable patients (72%). Platelet counts did not significantly vary according to H. pylori treatment outcome. Three of 56 patients (5%) achieved a partial response attributable to H. pylori eradication. Therefore, detection of H. pylori infection should not be routinely included in the initial work-up of ITP.
British Journal of Haematology 01/2002; 115(4):1002-3. · 4.94 Impact Factor
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R García-Sanz,
S Montoto,
A Torrequebrada,
A G de Coca,
J Petit,
A Sureda,
J A Rodríguez-García,
P Massó,
A Pérez-Aliaga,
M D Monteagudo, [......],
C Toledo,
A Alonso,
C Besses,
J Besalduch, I Jarque,
P Salama,
J A Rivas,
B Navarro,
J Bladé,
J F Miguel
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ABSTRACT: In this report we analyse the presenting features of a series of patients diagnosed with Waldenström macroglobulinaemia (WM) in Spain over the last 10 years. Criteria for diagnosis required a serum monoclonal IgM protein > or = 30 g/l and > 20% bone marrow lymphocytes. Two hundred and seventeen patients were included in the study, with a median age of 69 years and male/female ratio of 2:1. The most common symptoms at diagnosis were anaemia (38%), hyperviscosity (31%), B symptoms (23%), bleeding (23%) and neurological symptoms (22%). Sixty-one patients (27%) were asymptomatic at diagnosis and, to date, 32 of them have not received chemotherapy. Variables predicting a shorter survival free of therapy were haemoglobin < 12.5 g/dl and high beta2microglobulin (beta2M). The 83% of patients who did receive treatment were distributed as follows: chlorambucil/prednisone (43%), intermittent chlorambucil (11%), continuous chlorambucil (26%), cyclophosphamide/vincristine/prednisone (COP, 13.5%) and other (6.5%). Response to therapy was complete in 2%, partial in 48% and minor in 10%. Finally, 28% and 13% of patients presented stable and progressive disease, respectively, which was more common among patients treated with COP. Progression-free survival was 43% at 5 years, with three independent predictors for shorter progression-free survival (PFS): COP treatment, age > 65 and B symptoms at diagnosis. The 10-year projected overall survival (OS) was 55%. The two most frequent causes of death were development of second malignancies (31%), or infections (19%). The two main variables predicting a poor OS were hyperviscosity and high beta2M. In summary, this study favours the use of chlorambucil-based therapy as the standard treatment for WM, and describes a subset of patients who should be considered as suffering a smouldering form and who therefore do not require treatment for a long period of time.
British Journal of Haematology 12/2001; 115(3):575-82. · 4.94 Impact Factor
