BMT Patients Research Articles

18F-FDG PET Response After Induction Chemotherapy Can Predict Who Will Benefit from Subsequent Esophagectomy After Chemoradiotherapy for Esophageal Adenocarcinoma.

This study aimed to determine whether 18F-FDG PET response after induction chemotherapy before concurrent chemoradiotherapy can identify patients with esophageal adenocarcinoma who may benefit from subsequent esophagectomy. Methods: We identified and analyzed 220 patients with esophageal adenocarcinoma who had received induction chemotherapy before chemoradiotherapy, with or without surgery, with curative intent; all underwent 18F-FDG PET scanning before and after induction chemotherapy. 18F-FDG PET responders were defined as patients who achieved complete response (CR) after induction chemotherapy (maximum SUV ≤ 3.0). The predictive value of 18F-FDG PET response for patient outcomes was evaluated. Results: Overall, 86 patients had bimodality therapy (BMT; induction chemotherapy + chemoradiotherapy) and 134 had trimodality therapy (TMT; induction chemotherapy + chemoradiotherapy with surgery). Forty-eight patients (21.8%) achieved an 18F-FDG PET CR after induction chemotherapy. 18F-FDG PET CR was found to correlate with overall survival (OS) and progression-free survival (PFS) in BMT patients. For TMT patients, 18F-FDG PET CR predicted pathologic response (P = 0.003) but not survival. Among 18F-FDG PET nonresponders, TMT patients had significantly better survival than did BMT patients (P < 0.001). However, among 18F-FDG PET responders, BMT patients had OS (P = 0.201) and PFS (P = 0.269) similar to that of TMT patients. After propensity score-matched analysis, 18F-FDG PET responders treated with BMT versus TMT still had comparable OS and PFS, but TMT was associated with better locoregional control. Conclusion:18F-FDG PET response to induction chemotherapy could be a useful imaging biomarker to identify patients with esophageal adenocarcinoma who could benefit from subsequent esophagectomy after chemoradiotherapy. Compared with BMT, TMT can significantly improve survival in 18F-FDG PET nonresponders. However, outcomes for 18F-FDG PET responders were similar after either treatment (BMT or TMT). Prospective validation of these findings is warranted.

509 - Improving Sleep Quality of BMT Patients during the Inpatient Stay

509 - Improving Sleep Quality of BMT Patients during the Inpatient Stay

The Hospital at Home program: no place like home.

The treatment of children with cancer is associated with significant burden for the entire family. Frequent clinic visits and extended hospital stays can negatively affect quality of life for children and their families. Here, we describe the development of a Hospital at Home program (H@H) that delivers therapy to pediatric hematology, oncology, and blood and marrow transplant (bmt) patients in their homes. The services provided include short infusions of chemotherapy, supportive-care interventions, antibiotics, post-chemotherapy hydration, and teaching. From 2013 to 2015, the H@H program served 136 patients, making 1701 home visits, for patients mainly between the ages of 1 and 4 years. Referrals came from oncology in 82% of cases, from hematology in 11%, and from bmt in 7%. Since inception of the program, no adverse events have been reported. Family surveys suggested less disruption in daily routines and appreciation of specialized care by hematology and oncology nurses. Staff surveys highlighted a perceived benefit of H@H in contributing to early discharge of patients by supporting out-of-hospital monitoring and teaching. The development of a H@H program dedicated to the pediatric hematology, oncology, or bmt patient appears feasible. Our pilot program offers a potential contribution to improvement in patient quality of life and in cost-benefit for parents and the health care system.

Noninvasive versus invasive mechanical ventilation for immunocompromised patients with acute respiratory failure: a systematic review and meta-analysis.

BackgroundTo determine the effects of noninvasive mechanical ventilation (NIV) compared with invasive mechanical ventilation (IMV) as the initial mechanical ventilation on clinical outcomes when used for treatment of acute respiratory failure (ARF) in immunocompromised patients.MethodsWe searched PubMed, EMBASE, the Cochrane Central Register of Controlled Trials (CENTRAL), the Chinese Biomedical Literature Database (CBM) and other databases. Subgroup analyses by disease severity and causes of immunodeficiency were also conducted.ResultsThirteen observational studies with a total of 2552 patients were included. Compared to IMV, NIV was shown to significantly reduce in-hospital mortality (OR 0.43, 95 % CI 0.23 to 0.80, P value = 0.007) and 30-day mortality (OR 0.34, 95 % CI 0.20 to 0.61, P value < 0.0001) in overall analysis. Subgroup analysis showed NIV had great advantage over IMV for less severe, AIDS, BMT and hematological malignancies patients in reducing mortality and duration of ICU stay.ConclusionsThe overall evidence we obtained shows NIV does more benefits or at least no harm to ARF patients with certain causes of immunodeficiency or who are less severe.Electronic supplementary materialThe online version of this article (doi:10.1186/s12890-016-0289-y) contains supplementary material, which is available to authorized users.

Post-bone marrow transplant thrombotic microangiopathy.

Thrombotic microangiopathy (TMA) is a systemic disease characterized by microangiopathic hemolytic anemia, thrombocytopenia and organ failure. Post-bone marrow transplant TMA (post-BMT TMA) is a life-threatening condition that has been reported to afflict between 0.5 and 63.6% of BMT patients. The incidence of post-BMT TMA is affected by evolving therapies such as conditioning regimens. The etiology of post-BMT TMA is thought to be multifactorial, including the effects of immunosuppressive agents, viral infections, TBI and GvHD. A growing body of evidence highlights the importance of complement system activation and endothelial damage in post-BMT TMA. Although plasmapheresis has commonly been used, its therapeutic rationale for the majority of post-BMT TMA cases is unclear in the absence of circulatory inhibitors. It has become possible to target complement activation with eculizumab, a drug that blocks the terminal complement pathway. Early studies have highlighted the importance of anti-complement therapies in treating post-BMT TMA. Moreover, finding complement gene mutations may identify patients at risk, but whether such patients benefit from prophylactic anti-complement therapies before BMT remains to be studied. This review focuses on diagnostic criteria, pathophysiology, treatment and renal outcomes of post-BMT TMA.

The experiences of protective isolation in patients undergoing bone marrow or haematopoietic stem cell transplantation: systematic review and metasynthesis.

Protective isolation is aimed at preventing infection in neutropenic patients, but it is implemented inconsistently across centres and is supported by recommendations with poor evidence. This review and metasynthesis explored the experiences and the psychological implications of protective isolation in patients with haematological malignancies undergoing bone marrow (BMT) or haematopoietic stem cell transplantation (HSCT). A systematic search of multiple databases for qualitative studies exploring BMT or HSCT patients' experiences of protective isolation was completed. The metasynthesis followed the meta-aggregative method from the Joanna Briggs Institute, with four procedural steps: (1) comprehensive search, (2) quality appraisal, (3) extraction of relevant findings and (4) synthesis of the identified findings. Twenty-six findings were extracted from 11 articles included in the review. The synthesising process yielded seven categories, aggregated into three synthesised findings: (1) isolation is a source of suffering, (2) isolation can lead to relating with oneself and (3) the person does not close the door to the outside world. This metasynthesis sheds light on patients' suffering from being isolated, and the possibility of overcoming this suffering thanks to relationships that patients have with themselves and with the external world. Healthcare providers should reconsider this practise in order to avoid unnecessary patient suffering.

Integrative Therapy Use as a Comfort Measure with BMT Patients

Integrative Therapy Use as a Comfort Measure with BMT Patients

Quality Improvement: Decreasing Length of Stay, TPN and Filgrastim Usage in Autologous Stem Cell Transplant Patients

We used the Plan-Do-Study-Act quality improvement methodology to reduce the length of stay (LOS) for autologous stem cell transplant (ASCT) patients. Baseline data were collected from the electronic medical records of patients admitted for ASCT from January – April 2014 to serve as the control population. Changes in care were implemented during April 2014. The post implementation comparison intervals were phase I from May – October 2014 and phase II from November 2014 – April 2015 after re-education of staff. Changes in workflow included changing filgrastim administration post-stem cell infusion from day +6 to day +9. The utilization of TPN was changed from individual provider preferences to protocol based with a nutritional team member rounding with the inpatient hematology team. Criteria for starting TPN included patients with less than 50% required calories for >7 days, severe mucositis, or weight loss (>10%) from the time of hospital admission. TPN was not used within the first week of hospital admission unless clinically required. Time to neutrophil engraftment and LOS were monitored continuously during the study period. The pre-implementation median LOS was 17 days (n=16, range: 14-22 days) with a median neutrophil engraftment day of +12 (n=22, range: 10-13). Post-implementation with 3 days less of filgrastim, the median neutrophil engraftment day remained +12 (n=45, range: 10-14). Overall there was a decrease in the median LOS to 16 days (n=46, range: 14-29 days). The cost savings was approximately $1500 per patient due to the reduced filgrastim use and administration and approximately $8500 per patient for the decreased LOS. TPN use was 10% (n=20) at baseline. Post-implementation TPN use decreased to 8.33% (n=24) during phase I and 0% (n=23) during phase II. For the patients receiving TPN during phase I the start day went from +7.5 (pre-implementation) to day +11 and the average number of days on TPN decreased from 4.5 to 3 days. The cost savings was approximately $1000 per patient not requiring TPN. The implementation of protocol driven quality improvement parameters decreased our ASCT patients LOS by one day, decreased TPN use to 0% and decreased the need and cost associated with 3 days of filgrastim use. Re-education of providers at the beginning of phase II contributed to this continued improvement. These practice improvements have important clinical implications. Reduced LOS decreases infection risks and improves the quality of life (QOL) for patients and caregivers. Limiting TPN use also reduces the risks of infection and hepatic dysfunction. Decreased use of filgrastim decreases the risk of side effects including injection site reactions and bone pain, further improving patient QOL. Decreased LOS, TPN and filgrastim use, are all economically preferred and beneficial. An ongoing study is evaluating the same parameters in allogenic BMT patients.

Cytomegalovirus infection in children after bone marrow transplantation: Risk factors, clinical aspects and outcomes

Abstract Background and objectives Cytomegalovirus (CMV) infection remains the most common and potentially severe viral complication in patients given hematopoietic stem cell transplantation. The aim of this study was to determine the incidence, risk factors and outcomes of CMV infection in pediatric BMT unit. Material and methods This study was a retrospective analysis of clinical, laboratory and outcome data of 131 pediatric patients who underwent BMT. Results The mean age of the study population was 6.5 ± 4 years. Out of 131 pediatric patients, 85 were males (64.9%). Majority of patients had hematological disorder/malignancy ( n = 101; 77%) followed by solid tumors ( n = 30; 23%). Most of them received allogeneic transplant ( n = 92; 70.2%). CMV reactivation was observed in 38 (29%) patients; out of them, only ( n = 3; 2.3%) had clinical manifestation/organ involvement and most cases of CMV were resolved ( n = 35; 26.7%). Benign hematological disorder, conditioning regimen containing ATG, allogeneic BMT, graft-versus-host disease (GVHD) prophylaxis used and development of GVHD were identifiable risk factors in all patients, and lymphopenia 3 ( p = 0.047) was the only identifiable risk factors in allogeneic BMT patients associated with development CMV reactivation. Patients, who had CMV reactivation had significantly higher rate of GVHD (31.6% vs 15.1%; p = 0.031), however relapse rate (21% vs 25.8%) and mortality rate (22.5% vs 33.3%) in patients with CMV reactivation vs no CMV reactivation respectively were not statistically significant. Overall survival and event free survival of patients with and without CMV antigenemia were also comparable. Conclusions Antigenemia-guided pre-emptive strategy with ganciclovir was very affective and CMV reactivation tended not to affect the outcome in our study cohort.

Geriatric Assessment Metrics Are Associated with Hospital Length of Stay in Pre-Bone Marrow Transplant Myeloma Patients

A geriatric assessment (GA) is a global approach to improve healthy aging, wherein occult problems are assessed and intervened upon using a multidisciplinary method. A GA is feasible and can predict chemotherapy-induced toxicity and overall survival in cancer patients. Biomarkers of aging are also being explored as objective and reproducible measures of health and fitness. p16INK4a (p16) is a marker of cellular senescence that rises exponentially with chronologic age and is influenced by factors such as physical activity, smoking, and solid tumor chemotherapy. Here, we investigated the relationship of both the GA and molecular (i.e. p16) metrics in pre-bone marrow transplant (BMT) multiple myeloma (MM) patients. We selected this group for our studies as BMT patients are a vulnerable cohort in which transplant eligibility is subjective and age related. BMT patients are also at high risk for adverse events and treatment toxicity. In this preliminary analysis, we explored the predictive value of GA metrics and p16 with inpatient length of stay (LOS) during autologous BMT.Methods: We performed a pilot prospective cohort study on 55 MM patients during their pre-transplant evaluation. MM patients >18 years completed GA assessments related to physical function, distress, comorbidities, social support, and cognition. Patients completed surveys using the Brief Fatigue Inventory (BFI) (scale 1-10; moderate fatigue 4-6, severe fatigue 7+); Hospital Anxiety and Depression (HADS) (borderline case 8-10, definite case 11+); medical outcome study-social support survey (MOS-SSS) (scale 0-100, higher scores indicated greater support), Human Activity Profile (HAP) maximum activity score (MAS) and HAP-adjusted activity score (AAS), a 94-item questionnaire ranking tasks according to energy use validated in the BMT population, with higher scores indicating higher activity (Herzberg BBMT 2010). Objective measures of physical activity were measured using the Short Physical Performance Battery (SPPB) (range 0-12; impairment <9) and cognition was evaluated using the Modified Mini Mental Status exam (3MS). At the pre-transplant evaluation, p16 mRNA was measured in peripheral blood T-cells using established laboratory techniques (Liu Aging Cell 2009). The association between GA metrics and p16 were evaluated using Spearman's correlation coefficient. Univariable generalized linear models were fit to model LOS as a function of GA metrics or p16.Results: The median patient age was 61 (range 42-76). Most patients exhibited early stage disease (ISS Stage 1 53%) with minimal comorbidities (HCT-CI median 1; range 0-8) and a median of 2 prior lines of treatment (range 1-11). Pre-transplant Karnofsky Performance Status (KPS) was reported as 70% (n=10), 80% (n=10), 90% (n=15) and 100% (n=12). 7 patients did not proceed with BMT, 1 inpatient for BMT. Patients reported moderate fatigue by BFI (median 4.3; range 0-9.8), with minimal anxiety or depression as measured by the HADS. Self-reported physical activity by HAP-MAS was 73 (range 30-94) and HAP-AAS was 64 (range 20-94). Patients reported high levels of social support (median 86.7%; range 18.2-100) by MOS-SSS. Objective measures of physical function were also high as measured by the SPPB (median 10; range 4-12) and no cognitive impairment was identified by the 3MS. p16 expression was adjusted for age and did not correlate with GA tools including BFI, HADS, HAP-AAS, HAP-MAS, MOS-SSS, SPPB or 3MS. The median length LOS during transplant was 16 days (range 12-36). Univariate analysis revealed that SPPB score was significantly associated with LOS, where each one unit increase in physical performance corresponded to an average LOS decrease of 0.63 days (p=0.04). Self-reported activity by HAP-AAS also correlated with LOS (p=0.05). LOS was not influenced by p16, age, KPS or HCT-CI. Age and HCT-CI had no relationship with SPPB scores, but KPS did (p=0.03727).Conclusions: A comprehensive GA can be used to identify factors that contribute to BMT outcomes. Physical function appears to be most predictive of hospital LOS as measured by SPPB or a detailed self-report of physical function. Baseline p16 levels had no relationship with GA metrics in this selected population. A standardized approach for determining patient fitness including SPPB and HAP-AAS assessments may improve treatment tolerance, reduce hospital LOS, and decrease the risk for adverse outcomes in BMT populations. DisclosuresJaglowski:Immunomedics: Research Funding; Pharmacyclics LLC, an AbbVie Company: Consultancy, Research Funding; Seattle Genetics: Consultancy.

Embedded integration of palliative care in an academic outpatient cancer center.

158 Background: Palliative care is no longer reserved for ICUs and inpatient settings, since it can be delivered at any age and any stage regardless of goals of care. Oncologists are beginning to embrace more upstream palliative care but translating that to the long term setting has unique difficulties. Outpatient palliative care is a vital component to quality oncology care, but few examples of successful integration have been demonstrated. Methods: Data was collected from the palliative care clinic at the University of Kansas Medical Center Westwood Cancer Center. Descriptive analysis of the initial and current programs as well as successful outreach and education projects will be discussed. Quantitative analysis of number of initial and follow-up outpatient visits, cancer type, referring service and referring physician are all included. Hospice utilization was also recorded as well as length of time from initial consult to either death, discharge or calendar date June 30, 2015. Results: Since the pilot clinical program of outpatient palliative care in 2012 with two half day clinic sessions focused on BMT patients, the program has expanded to 5 half-day sessions and includes medical oncology, surgical oncology and gynecologic oncology. Referrals to palliative care have grown consistently covering multiple cancer diagnosis and including the entire oncology staff instead of a few champions. Increase in outpatient utilization has also spurred more early and appropriate inpatient palliative care utilization. Staff have moved from crisis-only use of outpatient palliative care to a co-management model. Conclusions: Outpatient palliative care can be successfully integrated into an NCI academic medical center oncology practice. (Note: More detailed data will be presented at the conference on all of the trends mentioned above.)

Combination Antifungal Therapy Experience at an Academic Health System

Invasive fungal infections are becoming increasingly prevalent, particularly in the immunocompromised population. The development of new antifungal agents with novel mechanisms of action has sparked interest in combination antifungal therapies. This study seeks to characterize combination antifungal use at a large academic health system. We conducted a retrospective observational study of adult inpatients with the intent of characterizing combination systemic antifungal utilization between the dates of July 1, 2012 and June 30, 2013. Combination antifungal therapy was defined as administration of two or more target systemic antifungals (liposomal amphotericin B, fluconazole, voriconazole, posaconazole, micafungin or terbinafine) for five or more days. Twenty nine patients received combination antifungal therapy and 36 unique treatment courses were identified. Ten of the 29 patients (35%) were BMT patients. Pneumonia was the most common disease indication (20/29; 69%). Unknown was the most common organism indication overall (11/29; 38%) and within the BMT patient population (6/10; 60%). Among the 36 unique treatment courses the most common combination therapy by drug class was an azole and an echinocandin (53%; 19/36) followed by a polyene and an azole (31%; 11/36). A significant proportion of the study population both overall (35%) and among BMT patients (60%) received combination antifungal therapy with no identified infecting fungal organism. There is no data to support such a practice. However, in the setting of severe invasive fungal infections, particularly in immunocompromised hosts, using additional antifungal agents may seem less risk-prone than invasive diagnostics like tissue biopsy. These findings suggest that earlier diagnostic methods including tissue biopsy are warranted to avoid unnecessary use of combination antifungal therapy when no pathogen has been identified.

Improving Transitions from the Inpatient to Outpatient Setting in BMT Patients

Improving Transitions from the Inpatient to Outpatient Setting in BMT Patients

Falls in BMT Patients

Falls in BMT Patients

Increasing Compliance in BMT Patients by Creating a Patient Education Binder

Newly diagnosed cancer patients with hematological disorders such as leukemia and lymphomas are for the most part diagnosed and quickly initiated on treatment. Information is constantly thrown at the patient from the moment he or she is diagnosed, often without being absorbed by the patient. These patients usually are initiated on chemotherapy and if warranted are headed to transplant. The patient education binder will allow the patient to collect all the information regarding disease and treatment in an organized manner divided into pertinent sections. Recognizing this is a stressful time, the binder will serve as a tool to ease anxiety. The binder will be started during the patient's initial diagnosis and follow the patient to end of recovery. The binder will contain useful information, which can assist patients during treatment. The nurses caring for the patient will review the material with the patient, checking off each section of the binder; thus nurses will be aware of any material needing reinforcement. Prior to initiating patient education, a small group of nurses were explained purpose of binder, division of binder, and checklist teachings. A small sample of newly diagnosed AML patients and transplant patients were chosen, and the binder was initiated at the beginning of treatment. A survey was conducted after patient was discharged regarding effect of the binder during patient’s stay. Outpatient nurses were also explained the purpose of the binder and importance of having the patient bring the binder to each visit. At this time no current patient education binders exist in the institution. There are other institutions where patient education binders are currently being used. Outcome measurement is easing of anxiety, compliance, and patient understanding of illness, medications, and treatment, assessed post discharge with a patient survey. Creating this binder could ultimately set forth a trend where all hospitals create a patient education binder to ease patient anxiety and increase compliance. Nurses and collaborative physicians can work together to ensure patient is compliant with binder, increasing patient education.

Happy Birthday…Again: Educating BMT Nurses and Patients about Infusion of Stem Cells

Happy Birthday…Again: Educating BMT Nurses and Patients about Infusion of Stem Cells

Ensuring Quality and Standardizing Care for BMT Patients in an Expanding Program

s / Biol Blood Marrow Transplant 21 (2015) S374eS390 S384 Findings & Interpretation: Since adoption of this new algorithm only 3 of 45 patients have experienced side-effects (P1⁄40.04). One patient had severe nausea, one had severe diarrhea and one experienced paresthesias. Discussion & Implications: The new dosing algorithm has not had a negative impact on stem cell mobilization. Standard doses of plerixafor can cause severe gastro-intestinal disturbance in patients with low body weight. Adjustment for body weight can prevent most of these side-effects.

The blood coagulation system and microcirculatory disorders in ixodid tick-borne borreliosis caused by Borrelia miyamotoi

To study blood coagulation and microcirculatory disorders as a possible cause of transient dysfunctions of organs (the kidney, liver, heart, lung, etc.) in patients with ixodid tick-borne borreliosis caused by Borrelia miyamotoi (Bmt). SUBJECTS AND METHODS; Twenty-four patients with Lyme disease (LD) and 28 Bmt patients treated at Izhevsk City Hospital (Udmurtia) were examined in the study. Platelet counts and the presence of D-dimers were determined; activated partial thromboplastin time, prothrombin time, thrombin time, fibrinogen and antithrombin III levels, and Factor XIIa-dependent fibrin clot lysis time were measured. Slit lamp microscopy of the conjunctiva was. also carried out. Results. Platelet counts'were less than 150,000 per pL of blood in 43% of the Bmt patients. All the Bmt patients had at least one abnormal coagulation parameter of the eight ones that were tested; 64% of them had marked coagulation disorders with three or more abnormal laboratory findings. In contrast, all the eight parameters were normal in 71% of the LD patients. The other seven LD patients had only one or two abnormal coagulation parameters (p < 0.001 in comparison with Bmt patients). Microscopic examination of eye capillary blood flow revealed pathological findings that included aggregates of erythrocytes and obstructed and/or sinuous capillaries in 22 (79%) of the Bmt patients, but none of the LD patients. A total of 14 Bmt patients had both coagulation and microcirculatory abnormalities. Eleven of them also had transient signs of organ dysfunction. As far as Borrelia secrete no known toxins, we hypothesized that uncovered disorders of blood coagulation and microcirculation in Bmt patients may contribute to organ dysfunction.

Distribution and Timing of Distant Metastasis after Local Therapy in a Large Cohort of Patients with Esophageal and Esophagogastric Junction Cancer

Background: Patients with localized esophageal and esophagogastric junction cancer (EAC) receive chemoradiation and then surgery (trimodality, TMT) or definitive chemoradiation (bimodality, BMT). Distant metastases (DMs) are common but the details of their distribution and timing in a large cohort have not been described. Methods: 629 patients with localized EAC who had TMT or BMT were analyzed. Standard statistical methods were used to define the end points. Results: The median follow-up time was 37.2 months (interquartile range 17.8-65.0). Of 356 TMT patients, 33% (119) developed DM as their first relapse and of 273 BMT patients, 40% (109) developed DM; 91% (TMT) and 96% (BMT) of the DMs were diagnosed within 2 years of local therapy. The most common sites of DM were: lung, distant nodes, liver, peritoneal cavity, bone, brain and pleura in order of frequency. The median overall survival of TMT patients with DM was 10.2 months (95% CI 7.8-12.7) and that for BMT patients with DM was 7.8 months (95% CI 5.7-9.9). Conclusions: Following TMT or BMT, ≥33% of patients developed DMs and most of these occurred within 2 years (>90%) of local therapy. A clinical model is desirable that associates clinical parameters with a high risk for DM in TMT-eligible patients prior to surgery.

Longitudinal Study on Liver Functions in Patients with Thalassemia Major before and after Deferasirox (DFX) Therapy.

By performing regular blood transfusion and iron chelation therapy, most patients with beta thalassemia major (BTM) now survive beyond the third decade of life. Liver disease is becoming an important cause of morbidity and mortality in these patients. Chronic hepatitis and/or severe iron overload are both important causes of liver pathology. Iron chelation with desferrioxamine (DFO) reduces excessive body iron, but its efficacy is limited by poor compliance and dose related toxicity. The recent use of Deferasirox ( DFX ), an oral single dose therapy, has improved the compliance to chelation.AimsTo study the long-term liver functions in BMT patients, seronegative for liver infections before versus after DFX treatment in relation to ferritin level.MethodsOnly BTM patients with hepatitis negative screening (checked every year) and on treatment with DFO for at least five years and with DFX for four years were enrolled. Liver function tests including serum bilirubin, alanine transferase (ALT), aspartate transferase (AST), albumin, insulin-like growth factor – I (IGF-I) and serum ferritin concentrations were followed every six months in 40 patients with BTM.ResultsDFX treatment (20 mg/kg/day) significantly decreased serum ferritin level in patients with BTM; this was associated with a significant decrease in serum ALT, AST, ALP and increase in IGF-I concentrations. Albumin concentrations did not change after DFX treatment. ALT and AST levels were correlated significantly with serum ferritin concentrations ( r = 0.45 and 0.33 respectively, p < 0.05). IGF-I concentrations were correlated significantly with serum ALT (r= 0.26, p = 0.05) but not with AST, ALP, bilirubin or albumin levels.The negative correlation between serum ferritin concentrations and ALT suggests that the impairment of hepatic function negatively affect IGF-I synthesis in these patients due to iron toxicity, even in the absence of hepatitis.ConclusionsSome impairment of liver function can occur in hepatitis negative thalassemic patients with iron overload. The use of DFX was associated with mild but significant reduction of ALT, AST and ALP and increase in IGF-I levels. The negative correlation between IGF-I and ALT concentrations suggest that preventing hepatic dysfunction may improve the growth potential in these patients.