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1.
Thromb Res ; 235: 92-97, 2024 Mar.
Article in English | MEDLINE | ID: mdl-38308884

ABSTRACT

INTRODUCTION: Atrial fibrillation or flutter (AF) is prevalent in cancer patients. Many of these patients have an indication for anticoagulation (AC) but are also at risk for developing chemotherapy-induced thrombocytopenia. There are scarce data regarding management of AC and risk of bleeding and thrombosis in cancer patients with AF and thrombocytopenia. AIM: To assess anticoagulation management and incidence of bleeding and arterial thromboembolism (ATE) in cancer patients with AF and grade 3-4 thrombocytopenia (platelets <50 × 109/L). METHODS: A retrospective cohort study included adults with active cancer, grade 3-4 thrombocytopenia and AF with CHA2DS2-VASc score ≥ 1. Patients were stratified according to AC discontinuation (No-AC) or continuation (Continue-AC) when platelets dropped below 50 × 109/L and followed for 30 days. The study outcomes were ATE (ischemic stroke, transient ischemic attack or systemic emboli) and major bleeding. Cox proportional hazards model was used to calculate hazard ratios (HR) with death as a competing risk (Fine and Gray model). RESULTS: The cohort included 131 patients; 90 in the No-AC group and 41 in the Continue-AC group. Patient characteristics were balanced between the groups. The 30-day cumulative incidence of ATE was 2 % [95 % CI 0.4 %-7 %] in the No-AC group and 2 % [0.2 %-11 %] in the Continue-AC group (HR 0.92 [95 % CI 0.09-9.88]). The 30-day cumulative incidence of major bleeding was 7.8 % [95 % CI 3.40 %-14.52 %] and 2.44 % [95 % CI 0.18 %-11.22 %] in the No-AC and Continue-AC groups, respectively (HR 3.29 [95 % CI 0.42-26.04]). CONCLUSIONS: The high rate of bleeding and low rate of ATE in thrombocytopenic cancer patients with AF suggests that holding AC during time-limited periods may be a reasonable approach.


Subject(s)
Atrial Fibrillation , Neoplasms , Thrombocytopenia , Adult , Humans , Atrial Fibrillation/complications , Atrial Fibrillation/drug therapy , Retrospective Studies , Thrombocytopenia/complications , Neoplasms/complications , Neoplasms/drug therapy , Hemorrhage/chemically induced , Anticoagulants/adverse effects
2.
PLoS One ; 19(2): e0292661, 2024.
Article in English | MEDLINE | ID: mdl-38324562

ABSTRACT

BACKGROUND: Venous thromboembolism (VTE) is considered a preventable cause of mortality. The evidence for the benefit of VTE prophylaxis in acute medical patients is non-conclusive. Meta-analysis of RCTs failed to demonstrate reduction of all-cause mortality, while showing higher risk of bleeding. The Israeli Ministry of Health has instructed to assess all acute medical patients for the risk for VTE using the Padua Prediction Score, without mandating prophylaxis. AIM: To evaluate the effect of filling the Padua score on clinical outcomes and VTE prophylaxis rates. METHODS: Retrospective Study was performed in Israel during the years 2014-2017. The participants were divided to Padua compliance vs non-compliance group. Primary outcome: 30-day mortality. Secondary outcomes: 90-day incidence of VTE and suspected major bleeding. A propensity-weighted logistic multiple regression was performed. RESULTS: 18,890 patients were included in the study. The fulfillment of the Padua score was associated with an increased use of VTE prophylaxis, OR 1.66 (95% CI 1.49-1.84). However, there was no reduction of mortality or VTE events, OR 1.13 (95% CI 0.97-1.31) and OR 1.22 (95% CI 0.79-1.8) respectively. Hospitalizations related to hemoglobin decrease were not statistically different between the two groups. CONCLUSIONS: Padua score for the assessment of VTE risk in medical wards was associated with higher administration of pharmacological prophylaxis without reduction in VTE or mortality rate. Its usage should be reassessed as a performance measure.


Subject(s)
Venous Thromboembolism , Humans , Retrospective Studies , Venous Thromboembolism/drug therapy , Anticoagulants/therapeutic use , Hospitalization , Hospitals , Hemorrhage/drug therapy , Risk Factors
3.
JAMA Oncol ; 10(1): 137-138, 2024 Jan 01.
Article in English | MEDLINE | ID: mdl-37991749

ABSTRACT

This cohort study compares the mortality and hospitalization risks among patients with vs without solid cancer and diagnosed with COVID-19 during the period when the Omicron variant was dominant.


Subject(s)
COVID-19 , Neoplasms , Humans , SARS-CoV-2 , Hospitalization
4.
Isr Med Assoc J ; 25(9): 585-589, 2023 Sep.
Article in English | MEDLINE | ID: mdl-37698307

ABSTRACT

BACKGROUND: Influenza and coronavirus disease 2019 (COVID-19) are respiratory diseases with similar modes of transmission. In December 2021, influenza re-emerged after it had been undetected since March 2020 and the Omicron variant replaced the Delta variant. Data directly comparing the two diseases are scarce. OBJECTIVES: To compare the outcomes of patients with both the Omicron variant and influenza during 2021-2022. METHODS: We performed a retrospective study conducted in Beilinson hospital, Israel, from December 2021 to January 2022. We included all hospitalized patients with either laboratory-confirmed COVID-19 or influenza. The primary outcome was 30-day mortality. RESULTS: We identified 167 patients diagnosed with Omicron and 221 diagnosed with Influenza A. The median age was 71 years for Omicron and 65 years for influenza. Patients with Omicron had a significantly higher Charlson Comorbidity Index score (4 vs. 3, P < 0.001). Patients with Omicron developed more respiratory failure that needed mechanical ventilation (7% vs. 2%, P = 0.05) and vasopressors (14% vs. 2%, P < 0.001) than patients with influenza. In a multivariate model, 30-day mortality was lower in patients diagnosed with influenza than in patients diagnosed with Omicron (19/221 [9%] vs. 44/167 [26%], hazard ratio 0.45, 95% confidence interval 0.25-0.81). CONCLUSIONS: Patients diagnosed with Omicron had higher mortality than patients diagnosed with seasonal influenza. This finding could be due to differences in co-morbidities, the virus pathogenicity, and host responses to infection.


Subject(s)
COVID-19 , Influenza, Human , Humans , Aged , Influenza, Human/epidemiology , COVID-19/epidemiology , COVID-19/therapy , Retrospective Studies , SARS-CoV-2 , Seasons
5.
Med. clín (Ed. impr.) ; 161(2): 59-61, jul. 2023. tab
Article in English | IBECS | ID: ibc-223275

ABSTRACT

Introduction Initiation of global vaccination significantly reduced the morbidity and mortality of COVID-19. During the Omicron wave, approximately 70% of the Israeli adult population was fully vaccinated, but the efficacy of the vaccine was questioned. Methods We conducted a retrospective cohort study of all adult patients admitted to the COVID-19 departments in Rabin Medical Center, during the Delta wave and the Omicron wave. Patients were matched in the 2 waves using the inverse probability of treatment weighting (IPTW) method and risk for mechanical ventilation and 30-day all-cause mortality was assessed. Results Vaccination had a significant effect on 30-day mortality in the Delta and Omicron waves with adjusted OR of 0.35 (0.17–0.70) and 0.5 (0.27–0.95) respectively. Nonetheless, the rate of mechanical ventilation was similar between the groups with OR of 0.75 (0.52–1.09) and 0.64 (0.40–1.01). Vaccination status did not change the length of admission in both waves. Conclusion We observed a decreased risk for 30-day mortality among vaccinated patients during the Delta and Omicron waves in Israel. This association, even though consistent, was of a lesser magnitude during the Omicron wave (AU)


Introducción La iniciación de la vacunación global redujo significativamente la morbilidad y la mortalidad de la COVID-19. Durante la ola de ómicron, aproximadamente 70% de la población adulta israelí estaba completamente vacunada, pero se cuestionó la eficacia de la vacuna. Métodos Realizamos un estudio de cohorte retrospectivo de todos los pacientes adultos ingresados en los departamentos de COVID-19 en el Centro Médico Rabin, durante las olas de delta y ómicron. Los pacientes fueron emparejados en las dos olas utilizando el método de ponderación inversa de probabilidad de tratamiento (IPTW) y se evaluó el riesgo de ventilación mecánica y la mortalidad por todas las causas a los 30 días. Resultados La vacunación tuvo un efecto significativo en la mortalidad a los 30 días en las olas de delta y ómicron con odds ratio (OR) ajustadas de 0,35 (0,17-0,70) y 0,5 (0,27-0,95), respectivamente. Sin embargo, la tasa de ventilación mecánica fue similar entre los grupos con OR de 0,75 (0,52-1,09) y 0,64 (0,40-1,01). El estado de vacunación no cambió la duración del ingreso en ambas olas.Conclusión Observamos un menor riesgo de mortalidad a los 30 días entre los pacientes vacunados durante las olas de delta y ómicron en Israel. Esta asociación, aunque constante, fue de menor magnitud durante la ola de ómicron (AU)


Subject(s)
Humans , Male , Female , Middle Aged , Aged , Aged, 80 and over , Coronavirus Infections/prevention & control , Pneumonia, Viral/prevention & control , Viral Vaccines , Severity of Illness Index , Coronavirus Infections/mortality , Pneumonia, Viral/mortality , Retrospective Studies , Cohort Studies , Israel
6.
Med Clin (Barc) ; 161(2): 59-61, 2023 07 21.
Article in English, Spanish | MEDLINE | ID: mdl-37248130

ABSTRACT

INTRODUCTION: Initiation of global vaccination significantly reduced the morbidity and mortality of COVID-19. During the Omicron wave, approximately 70% of the Israeli adult population was fully vaccinated, but the efficacy of the vaccine was questioned. METHODS: We conducted a retrospective cohort study of all adult patients admitted to the COVID-19 departments in Rabin Medical Center, during the Delta wave and the Omicron wave. Patients were matched in the 2 waves using the inverse probability of treatment weighting (IPTW) method and risk for mechanical ventilation and 30-day all-cause mortality was assessed. RESULTS: Vaccination had a significant effect on 30-day mortality in the Delta and Omicron waves with adjusted OR of 0.35 (0.17-0.70) and 0.5 (0.27-0.95) respectively. Nonetheless, the rate of mechanical ventilation was similar between the groups with OR of 0.75 (0.52-1.09) and 0.64 (0.40-1.01). Vaccination status did not change the length of admission in both waves. CONCLUSION: We observed a decreased risk for 30-day mortality among vaccinated patients during the Delta and Omicron waves in Israel. This association, even though consistent, was of a lesser magnitude during the Omicron wave.


Subject(s)
COVID-19 , Adult , Humans , Israel/epidemiology , COVID-19/prevention & control , Retrospective Studies , Hospitalization , Hospitals
7.
Medicine (Baltimore) ; 102(6): e32812, 2023 Feb 10.
Article in English | MEDLINE | ID: mdl-36820599

ABSTRACT

Patients with inflammatory bowel disease (IBD) have a higher incidence of Clostridioides difficile infection (CDI). Previous studies have demonstrated negative clinical outcomes in IBD patients with CDI compared to patients without CDI. The clinical presentation of CDI is indistinguishable from IBD exacerbation, thus posing a frequent clinical dilemma on the role of Clostridioides infection in the testing, diagnosis, and treatment of these patients. To compare clinical outcomes of CDI in patients with IBD to those without IBD. Retrospective cohort of adult patients admitted to Rabin Medical Center Israel between the years 2014 and 2020 with a concurrent diagnosis of IBD and CDI. Matching 1:2 was performed between the IBD patients and the non-IBD population with respect to age and sex. Sixty-seven patients with IBD and 134 patients without IBD were included in the study. The groups' median age was 40.6 (interquartile range [IQR] of 29.8-68.9), with 45.8% male and 54.2% female. The non-IBD group had a higher Charlson score with 2 (IQR 0; 5) versus 0 (IQR 0; 4) in the IBD group (P value <.01). Patients with IBD had more exposure to systemic antibiotics, 71.1% versus 26.3% (P value <.01). In a multivariable analysis we found no difference in 90-day mortality and rate of relapse between the 2 study groups with an odds ratio of 1.709 (95% confidence interval 0.321-9.905) and odds ratio of 0.209 (95% confidence interval 0.055-1.513) respectively. In our cohort patients with IBD who present with diarrhea and concomitant CDI have similar rates of relapse and mortality compared with patients without IBD.


Subject(s)
Clostridioides difficile , Clostridium Infections , Inflammatory Bowel Diseases , Adult , Humans , Male , Female , Retrospective Studies , Clostridioides , Risk Factors , Inflammatory Bowel Diseases/complications , Inflammatory Bowel Diseases/epidemiology , Inflammatory Bowel Diseases/drug therapy , Clostridium Infections/complications , Clostridium Infections/drug therapy , Clostridium Infections/epidemiology , Diarrhea/epidemiology , Diarrhea/complications , Recurrence
8.
J Thromb Thrombolysis ; 52(2): 590-596, 2021 Aug.
Article in English | MEDLINE | ID: mdl-33523384

ABSTRACT

Managing anticoagulation in hematological malignancy patients with atrial fibrillation and thrombocytopenia is a clinical challenge with limited data. We aimed to identify anticoagulation management strategies and evaluate bleeding and thrombosis rates associated with each approach. A retrospective cohort study in Israel and the Netherlands was conducted. Patients with hematological malignancy and atrial fibrillation were indexed when platelets were < 50 × 109/L and followed for 30 days. The cohort included 61 patients of whom 42 (69%) had anticoagulation held at index. On multivariate analysis, holding anticoagulation was associated with age < 65 years and atrial fibrillation diagnosed within 30 days prior index. Clinically relevant bleeding was diagnosed in 7 (16.7%) and 1 (5.3%) of patients who had anticoagulation held and continued respectively, while arterial thromboembolism occurred in 1 patient in each group (2.4% and 5.3%, respectively). All-cause mortality rate was high at 45%. Accordingly, the 30-day bleeding risk may outweigh the risk of arterial thromboembolism in hematological malignancy, platelets < 50 × 109/L and atrial fibrillation.


Subject(s)
Anemia , Atrial Fibrillation , Hematologic Neoplasms , Thrombocytopenia , Thromboembolism , Aged , Anticoagulants/adverse effects , Atrial Fibrillation/complications , Atrial Fibrillation/drug therapy , Hematologic Neoplasms/complications , Hematologic Neoplasms/drug therapy , Hemorrhage/chemically induced , Humans , Retrospective Studies , Thrombocytopenia/chemically induced , Thrombocytopenia/drug therapy
9.
Microb Drug Resist ; 27(3): 410-414, 2021 Mar.
Article in English | MEDLINE | ID: mdl-32808858

ABSTRACT

Objective: Carbapenems are considered treatment of choice for bacteremia caused by potential AmpC-producing bacteria, including Enterobacter spp. We aimed to compare mortality following carbapenem vs. alternative antibiotics for the treatment of Enterobacter spp. bacteremia. Patients and Methods: We conducted a retrospective study in two centers in Israel. We included hospitalized patients with Enterobacter bacteremia treated with third-generation cephalosporins (3GC), piperacillin/tazobactam, quinolones, or carbapenem monotherapy as the main antibiotic in the first week of treatment, between 2010 and 2017. Cefepime was excluded due to nonavailability during study years. The primary outcome was 30-day all-cause mortality. Univariate and multivariate analyses were conducted, introducing the main antibiotic as an independent variable. Results: Two hundred seventy-seven consecutive patients were included in the analyses. Of these, 73 were treated with 3GC, 39 with piperacillin/tazobactam, 104 with quinolones, and 61 with carbapenems. All-cause 30-day mortality was 16% (45 patients). The type of antibiotics was not significantly associated with mortality on univariate or multivariate analyses. With carbapenems as reference, adjusted odds ratios (ORs) for mortality were 0.708, 95% confidence interval (CI) 0.231-2.176 with 3GC; OR 1.172, 95% CI 0.388-3.537 with piperacillin/tazobactam; and OR 0.586, 95% CI 0.229-1.4 with quinolones. The main antibiotic was not associated with repeated growth of Entrobacter spp. in blood cultures or other clinical specimens. Resistance development was observed with 3GC and piperacillin/tazobactam. Conclusions: Carbapenem treatment was not advantageous to alternative antibiotics, including 3GC, among patients with Enterobacter spp. bacteremia in an observational study.


Subject(s)
Anti-Bacterial Agents/therapeutic use , Bacteremia/drug therapy , Bacteremia/microbiology , Bacteremia/mortality , Enterobacter/drug effects , Aged , Aged, 80 and over , Anti-Bacterial Agents/pharmacology , Carbapenems/pharmacology , Carbapenems/therapeutic use , Female , Humans , Israel , Lymphokines , Male , Microbial Sensitivity Tests , Middle Aged , Peptides, Cyclic , Piperacillin, Tazobactam Drug Combination/pharmacology , Piperacillin, Tazobactam Drug Combination/therapeutic use , Quinolones/pharmacology , Quinolones/therapeutic use , Retrospective Studies , Socioeconomic Factors
10.
Am J Med ; 133(12): 1444-1452.e3, 2020 12.
Article in English | MEDLINE | ID: mdl-32445719

ABSTRACT

OBJECTIVE: Current guidelines recommend pharmacologic prophylaxis for medical patients at high risk for venous thromboembolism. We aimed to assess the benefit and safety of venous thromboembolism prophylaxis in acutely ill medical patients hospitalized. METHODS: We prepared a retrospective cohort study in a tertiary hospital in Israel with patients hospitalized in medical departments with an admission lasting more than 48 hours during 2014-2017. PRIMARY OUTCOME: 30-day mortality. SECONDARY OUTCOMES: 90-day incidence of pulmonary embolism, symptomatic deep vein thrombosis, and major bleeding. Propensity-weighted logistic multivariate analysis was performed. RESULTS: A total of 18,890 patient-unique episodes were included in the analysis. Of them, 3206 (17.0%) received prophylaxis. A total of 1309 (6.9%) died, 540/3206 (16.8%) of those who received venous thromboembolism prophylaxis and 769/15,864 (4.9%) of those who did not. Prophylaxis was not associated with a reduction in mortality, multivariable-adjusted odds ratio propensity-weighted (OR) 0.99 (95% confidence interval [CI], 0.84-1.14). One hundred forty-two patients (0.7%) developed venous thromboembolism, 44/3206 (1.4%) of those who received prophylaxis and 98/15,864 (0.6%) of those who did not. Prophylaxis was not associated with reduction in venous thromboembolism in the whole cohort, multivariable-adjusted propensity-weighted OR 1.09 (95% CI, 0.52-2.29). Prophylaxis was associated with an increase in major bleeding (multivariable-adjusted propensity-weighted OR 1.24; 95% CI, 1.04-1.48). CONCLUSION: The current practice of routinely administering venous thromboembolism prophylaxis to medically ill patients considered at high risk for thrombosis resulted in a high risk for bleeding without a clear clinical benefit, and should be reassessed.


Subject(s)
Anticoagulants/adverse effects , Anticoagulants/therapeutic use , Hemorrhage/prevention & control , Venous Thromboembolism/prevention & control , Cohort Studies , Humans , Odds Ratio , Retrospective Studies , Risk Factors
12.
Diagn Microbiol Infect Dis ; 93(2): 120-124, 2019 02.
Article in English | MEDLINE | ID: mdl-30241971

ABSTRACT

Use of GeneXpert MRSA/SA in diagnostic algorithms of Staphylococcus aureus bacteremia may influence both patients' clinical outcomes and antibiotic stewardship. We evaluated these outcomes in a retrospective cohort before (1/6/2015-31/5/2016) and after (1/6/2016-31/8/2017) the introduction of the test in adult patients with Gram-positive cocci in clusters in blood cultures. We included 254 patients (125 preintervention, 129 postintervention). No significant difference in 30-day mortality or clinical success was demonstrated between periods. Appropriate antibiotic therapy rates were significantly higher in the postintervention group, and vancomycin use was significantly reduced (80.6% vs 53.6%, P < 0.01; 2.3±0.38 vs 2.98±1.02 defined daily doses/100 patient days, P = 0.026, respectively). Appropriate beta-lactam use was also significantly higher (56.7% postintervention vs 23.1% preintervention, P < 0.01). Use of GeneXpert MRSA/SA test has a positive effect on antibiotic stewardship measures, though it has no significant effect on clinical outcomes including mortality in this fatal infection.


Subject(s)
Bacteremia/diagnosis , Bacteremia/mortality , Methicillin-Resistant Staphylococcus aureus/genetics , Molecular Typing , Staphylococcal Infections/diagnosis , Staphylococcal Infections/mortality , Aged , Aged, 80 and over , Bacteremia/drug therapy , Bacteremia/microbiology , Female , Humans , Male , Middle Aged , Molecular Typing/methods , Molecular Typing/statistics & numerical data , Polymerase Chain Reaction/methods , Polymerase Chain Reaction/statistics & numerical data , Retrospective Studies , Staphylococcal Infections/drug therapy , Staphylococcal Infections/microbiology , Staphylococcus aureus/genetics
13.
Eur J Gastroenterol Hepatol ; 30(6): 608-611, 2018 06.
Article in English | MEDLINE | ID: mdl-29384794

ABSTRACT

BACKGROUND: During the Ramadan fast Muslims celebrate a month of prayer, dawn-to-dusk fasting, and nightly feasts. We aimed to assess whether acute pancreatitis (AP) is more common during the Ramadan fast in individuals who celebrate it. PATIENTS AND METHODS: The study was carried out at the emergency department of Rabin Medical Center, Israel. We compared the occurrence of AP in a fasting population and a nonfasting population during the Ramadan versus the rest of the year. RESULTS: Over the 10-year study period, 1167 patients were admitted to the emergency department with AP. Of these, 1069 (91.6%) were nonfasting and 98 (8.4%) were fasting. Of these, 17/98 (17.3%) fasting patients and 95/1069 (8.8%) nonfasting patients were admitted with AP during the Ramadan [relative risk: 1.12; 95% confidence interval (CI): 1.004-1.2; odds ratio (OR): 2.15; 95% CI: 1.23-3.8; P=0.01]. During the Ramadan, the rate of AP out of referrals was 0.1% (17/15 068) in fasting patients versus 0.004% (95/213 913) in nonfasting individuals (OR: 2.54; 95% CI: 1.5-4.25). During the other months of the year, the rate of AP out of referrals was 0.009% (81/86 072) in fasting patients versus 0.008% (974/1 202 405) in nonfasting individuals (OR: 1.16; 95% CI: 0.92-1.45; P<0.001). CONCLUSION: We found a high rate of AP in the fasting population during the Ramadan. Physicians should be aware of this link and suspect it for fasting patients presenting with epigastric pain during the Ramadan fast.


Subject(s)
Fasting , Islam , Pancreatitis/epidemiology , Acute Disease , Adolescent , Adult , Aged , Aged, 80 and over , Databases, Factual , Female , Humans , Israel/epidemiology , Male , Middle Aged , Odds Ratio , Pancreatitis/diagnosis , Prevalence , Retrospective Studies , Risk Factors , Time Factors , Young Adult
15.
J Thromb Thrombolysis ; 44(3): 371-375, 2017 Oct.
Article in English | MEDLINE | ID: mdl-28748519

ABSTRACT

Antiphospholipid syndrome (APS) is classified as the association of a thrombotic event and/or obstetric morbidity in patients persistently positive for antiphospholipid antibodies and/or lupus anticoagulant. To evaluate the incidence of subsequent thrombosis among women diagnosed with purely obstetric APS. We retrospectively reviewed and collected demographic and clinical data from the computerized charts of all patients with obstetric APS, from 1992 to 2017. Eligibility criteria included all women diagnosed with APS, according to the 2006 revised criteria, for whom the clinical manifestations were purely obstetric. The primary endpoint was the occurrence of subsequent thromboembolic events, following diagnosis of obstetric APS. The study included 115 women diagnosed with obstetric APS. During the study's follow up period, 12 (10.4%) women developed thrombosis. Of the 12 women who developed thrombosis, 9 (75%) of the thrombotic events were arterial. The site of arterial thrombosis was cerebral in all cases. Venous thrombosis occurred in 3 (25%) women, including one in each of the following sites-pulmonary embolism, ovarian vein thrombosis and proximal leg deep vein thrombosis. Our data suggests that women with obstetric APS are at risk for subsequent long-term thrombosis, especially arterial cerebral events. We did not identify any clinical or laboratory unique features among women with obstetric APS who will eventually develop thrombosis.


Subject(s)
Antiphospholipid Syndrome/complications , Pregnancy Complications , Thrombosis/etiology , Adult , Female , Humans , Intracranial Thrombosis , Pregnancy , Retrospective Studies , Time Factors , Venous Thrombosis , Young Adult
16.
Pediatr Blood Cancer ; 63(3): 535-40, 2016 Mar.
Article in English | MEDLINE | ID: mdl-26485304

ABSTRACT

BACKGROUND: Hematopoietic stem cell transplantation (HSCT) is the only curative treatment for infantile malignant osteopetrosis (IMO), but is associated with a high incidence of adverse outcomes. In this study, we present our experience with HSCT for IMO patients comparing different types of conditioning regimens. METHODS: Thirty-eight patients with IMO (aged from 1 month to 6 years, median 0.66 years) who underwent allogeneic HSCT from 1983 in our hospital were included in this retrospective study. Fludarabine-based conditioning regimens were used in 26 patients and 12 patients were transplanted using other conditioning regimens. RESULTS: The overall survival after conditioning with fludarabine was 96% (25/26) versus 58% (7/12) for the alternative regimens (P = 0.004), with significantly fewer adverse effects including hypercalcemia and veno-occlusive disease of liver. All patients who survive are clinically well. CONCLUSIONS: We conclude that fludarabine-based conditioning regimens are safe and effective in patients with IMO, improving morbidity and mortality related to HSCT.


Subject(s)
Hematopoietic Stem Cell Transplantation , Osteopetrosis/therapy , Transplantation Conditioning/methods , Vidarabine/analogs & derivatives , Disease-Free Survival , Female , Graft vs Host Disease/prevention & control , Hepatic Veno-Occlusive Disease/etiology , Humans , Hypercalcemia/etiology , Infant , Infant, Newborn , Male , Osteopetrosis/mortality , Postoperative Complications , Tissue Donors , Tissue Survival , Treatment Outcome , Vidarabine/pharmacology
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