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Background In Mexico, patients with hematologic malignancies (HMs) are characterized by being at high risk and advanced stages at diagnosis and by having a low cure rate; yet information on their experiences with health care and health-related quality of life (HRQL) is scarce. We aimed to evaluate experiences with health care and HRQL of patients with HMs and the association between these patient-reported measures. Methods We conducted a cross-sectional survey in two public oncology hospitals in Mexico City. The study included outpatient cancer patients aged ≥18 years with a diagnosis of leukemia, lymphoma, or multiple myeloma. We used a patient-centered quality of cancer care questionnaire to assess patient experiences with receiving 1) timely care; 2) clear information; 3) information for treatment decision-making; 4) care to address biopsychosocial needs; and 5) respectful and coordinated care. We applied the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) to measure HRQL. We performed a multiple linear regression to evaluate the association between patient-reported experiences (independent variables) and the QLQ-C30 summary score (dependent variable). Results Of the 515 participating HM patients, 46.6% had lymphoma, 34% leukemia, and 19.4% multiple myeloma; 70.9% were at advanced stages or at high risk. Additionally, 15.1% had anxiety and 12.8% had depression. Over one third (35.9%) reported receiving clear information, 28.5% timely care, 20.6% information for treatment decision-making, 23.7% care that addressed their biopsychosocial needs, and 31% respectful and coordinated care. The mean QLQ-C30 summary score was 71.9 points. Timely care, clear information, and care that addresses biopsychosocial needs were associated with higher HRQL. Conclusions Health care services for HM patients at public oncology hospitals in Mexico need improvement. Notably, providing timely care, clear information, and care that addresses patients’ biopsychosocial needs can increase the likelihood of better HRQL. Health care providers should measure and improve the experiences of HM patients with health care.

Background: In Mexico, patients with hematologic malignancies (HMs) are characterized by being at high risk and advanced stages at diagnosis and by having a low cure rate; yet information on their experiences with health care and health-related quality of life (HRQL) is scarce. We aimed to evaluate experiences with health care and HRQL of patients with HMs and the association between these patient-reported measures. Methods: We conducted a cross-sectional survey in two public oncology hospitals in Mexico City. The study included outpatient cancer patients aged ≥18 years with a diagnosis of leukemia, lymphoma, or multiple myeloma. We used a patient-centered quality of cancer care questionnaire to assess patient experiences with receiving 1) timely care; 2) clear information; 3) information for treatment decision-making; 4) care to address biopsychosocial needs; and 5) respectful and coordinated care. We applied the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) to measure HRQL. We performed a multiple linear regression to evaluate the association between patient-reported experiences (independent variables) and the QLQ-C30 summary score (dependent variable). Results: Of the 515 participating HM patients, 46.6% had lymphoma, 34% leukemia, and 19.4% multiple myeloma; 70.9% were at advanced stages or at high risk. Additionally, 15.1% had anxiety and 12.8% had depression. Over one third (35.9%) reported receiving clear information, 28.5% timely care, 20.6% information for treatment decision-making, 23.7% care that addressed their biopsychosocial needs, and 31% respectful and coordinated care. The mean QLQ-C30 summary score was 71.9 points. Timely care, clear information, and care that addresses biopsychosocial needs were associated with higher HRQL. Conclusions: Health care services for HM patients at public oncology hospitals in Mexico need improvement. Notably, providing timely care, clear information, and care that addresses patients’ biopsychosocial needs can increase the likelihood of better HRQL. Health care providers should measure and improve the experiences of HM patients with health care.

Taliglucerase alfa is an intravenous enzyme replacement therapy approved for treatment of type 1 Gaucher disease (GD), and is the first available plant cell-expressed recombinant therapeutic protein. Herein, we report long-term safety and efficacy results of taliglucerase alfa in treatment-naïve adult patients with GD. Patients were randomized to receive taliglucerase alfa 30 or 60 U/kg every other week, and 23 patients completed 36 months of treatment. Taliglucerase alfa (30 U/kg; 60 U/kg, respectively) resulted in mean decreases in spleen volume (50.1%; 64.6%) and liver volume (25.6%; 24.4%) with mean increases in hemoglobin concentration (16.0%; 35.8%) and platelet count (45.7%; 114.0%), and mean decreases in chitotriosidase activity (71.5%; 82.2%). All treatment-related adverse events were mild to moderate in intensity and transient. The most common adverse events were nasopharyngitis, arthralgia, upper respiratory tract infection, headache, pain in extremity, and hypertension. These 36-month results of taliglucerase alfa in treatment-naïve adult patients with GD demonstrate continued improvement in disease parameters with no new safety concerns. These findings extend the taliglucerase alfa clinical safety and efficacy dataset. www.clinicaltrials.gov identifier NCT00705939. Am. J. Hematol. 91:656-660, 2016. © 2016 Wiley Periodicals, Inc.

Taliglucerase alfa, the first available plant cell–expressed recombinant therapeutic protein, is an enzyme replacement therapy approved for Gaucher disease (GD). PB-06-001, a pivotal phase 3, multicenter, randomized, double-blind, parallel-dose study investigated taliglucerase alfa 30 or 60U/kg every other week through 9months in treatment-naïve adults with GD; 30-month extension study PB-06-003 followed. Patients completing PB-06-001 and PB-06-003 could continue treatment in PB-06-007. Nineteen patients enrolled in PB-06-007 (30U/kg, n=8; 60U/kg, n=9; dose adjusted, n=2); 17 completed 5 total years of treatment. In these 3 groups, respectively, taliglucerase alfa resulted in mean decreases in spleen volume (−8.7, −6.9, −12.4 multiples of normal), liver volume (−0.6, −0.4, −0.5 multiples of normal), chitotriosidase activity (−83.1%, −93.4%, −87.9%), and chemokine (CC motif) ligand 18 concentration (−66.7%, −83.3%, −78.9%), as well as mean increases in hemoglobin concentration (+2.1, +2.1, +1.8mg/dL) and platelet count (+31,871, +106,800, +34,000/mm3). The most common adverse events were nasopharyngitis and arthralgia. Most adverse events were mild/moderate; no serious adverse events were considered treatment-related. These results demonstrate continued improvement of disease parameters during 5years of taliglucerase alfa therapy in 17 treatment-naive patients with no new safety concerns, extending the taliglucerase alfa clinical efficacy and safety dataset. This study was registered at www.clinicaltrials.gov as NCT01422187.

On page 5767 in the 24 November 2011 issue, there are errors in the affiliations. The affiliation of the eleventh author (Heitner) is misspelled. The correct affiliation is University of Witwatersrand. The affiliation of the last author (Aviezer) is incomplete. His academic affiliation, Faculty of

Taliglucerase alfa (Protalix Biotherapeutics, Carmiel, Israel) is a novel plant cell–derived recombinant human β-glucocerebrosidase for Gaucher disease. A phase 3, double-blind, randomized, parallel-group, comparison-dose (30 vs 60 U/kg body weight/infusion) multinational clinical trial was undertaken. Institutional review board approvals were received. A 9-month, 20-infusion trial used inclusion/exclusion criteria in treatment-naive adult patients with splenomegaly and thrombocytopenia. Safety end points were drug-related adverse events: Ab formation and hypersensitivity reactions. Primary efficacy end point was reduction in splenic volume measured by magnetic resonance imaging. Secondary end points were: changes in hemoglobin, hepatic volume, and platelet counts. Exploratory parameters included biomarkers and bone imaging. Twenty-nine patients (11 centers) completed the protocol. There were no serious adverse events; drug-related adverse events were mild/moderate and transient. Two patients (6%) developed non-neutralizing IgG Abs; 2 other patients (6%) developed hypersensitivity reactions. Statistically significant spleen reduction was achieved at 9 months: 26.9% (95% confidence interval [CI]: −31.9, −21.8) in the 30-unit dose group and 38.0% (95% CI: −43.4, −32.8) in the 60-unit dose group (both P < .0001); and in all secondary efficacy end point measures, except platelet counts at the lower dose. These results support safety and efficacy of taliglucerase alfa for Gaucher disease. This study was registered at www.clinicaltrials.gov as NCT00376168.

We report here a case of pulmonary aspergilloma in a patient with acute myeloblastic leukemia. We present the clinical and radiological characteristics of this case. Aspergillus flavus was isolated. The antifungal treatment was unsuccessful. Pulmonary aspergillomas are associated with a high mortality in the immunocompromised host. It is important to know the diagnostic approach and the surgical and medical treatment of this disease.