Increased Incidence of Infection in Patients with Myelofibrosis and Transfusion-Associated Iron Overload: A Monocentric Experience

Background. Transfusion-associated iron overload may increase the risk of infections both increasing bacterial or fungal growth and leading to the production of free iron that impairs immune system response. Anemia and transfusion dependency (TD) represent well known prognostic factors of survival in patients with myelofibrosis (MF). Compared to myelodysplastic syndrome (MDS), the role of iron overload on infection in MF has been scarcely explored. Methods. We identified consecutive adult patients diagnosed at our Centre with primary or secondary MF, between 1998 and 2018. Patients were stratified in 2 groups according to transfusion dependence defined as having received >2 unit of red blood cells (RBC) over 3 months. The total number of RBC units infused was also collected. All infections were recorded and graded according to Common Terminology Criteria for Adverse Events (CTCAE). Results. A total of 106 patients, median age 72 years (range 44-89) were retrospectively evaluated. A diagnosis of primary MF was performed in 75 of cases (71%), post Essential Thrombocythemia myelofibrosis (PET-MF) in 27 (25%) and post Polycythemia Vera myelofibrosis (PPV-MF) in 4 (4%). Splenomegaly was present in 83 patients (78%) and 30 (28%) reported constitutional symptoms.According to the IPSS, 43 patients (40 %) were classified at low or intermediate-1 risk, and 63 (60 %) at intermediate-2 or high risk. Molecular analysis was performed in 76 patients (72%): 54 of 76 patients (71%) were positive for the JAK2V617F mutation, 10 (13%) for CALR mutation, 4 (5%) for MPL, and 8 (11%) were negative for all tested mutations. Over time, 56 patients (53%) received hydroxyurea or other cytoreductive treatment, and 23 (22%) ruxolitinib. Overall, 39 patients (37%) were transfusion dependent with a median of 14 RBC units received during follow-up (range 4-199). The median serum ferritin level in the TD cohort was 840 ng/mL (range 130-12.129). Median observation time was 36 months (range 3-203). At last follow-up, 48 patients (45%) presented one or more infectious episodes. Total infectious events were 69 and 13 of them (19%) were defined as severe (grade 3-4 CTCAE). Anatomical site of infections was the respiratory tract in 28 (41%) of cases, gastro-intestinal in 22 (32%), gynecological-urological in 8 (11%), sepsis in 2 (3%), unspecified in the remaining cases. When detected, the etiological agents were bacterial in 8 (12%), viral in 5 (7%) and fungal in 4 (6%). The 60-month cumulative incidence of infection from diagnosis of MF was 64.1±6.5%. TD patients showed a higher incidence of infection (99±8.8% vs 53.3±8%, p=0.007) (Figure 1). In multivariate analysis no association was found between infection incidence and primary vs secondary MF, splenomegaly, age >65 years, ruxolitinib treatment; only transfusion dependencemaintained a significant association (p=0.019; HR=2.13; 95% C.I.=1.13-4.01). Among the 39 TD patients, the transfusion burden was significantly higher in those with infectious complication (median 24 RBC units vs 15 RBC units; p=0.012). The 60-month overall survival was 40±5.9%. Lower IPSS-risk (p<0.0001) and ruxolitinib treatment (p=0.027) were significantly associated with higher survival. Conclusions. This retrospective monocentric real-life study showed an increased risk of infections in MF patients with higher transfusion burden. The role of iron chelation in improving infection free survival in patients with MF and iron overload should be further investigated. Figure 1 Disclosures No relevant conflicts of interest to declare.

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Prognostic role of neoplastic markers in Takotsubo syndrome

Scientific Reports ◽

10.1038/s41598-021-95990-9 ◽

2021 ◽

Vol 11 (1) ◽

Author(s):

Francesco Santoro ◽

Tecla Zimotti ◽

Adriana Mallardi ◽

Alessandra Leopizzi ◽

Enrica Vitale ◽

...

Keyword(s):

Serum Levels ◽

Takotsubo Syndrome ◽

Ca 15.3 ◽

Risk Of Death ◽

Increased Risk ◽

Ca 19.9 ◽

Long Term Follow Up

AbstractTakotsubo syndrome (TTS) is an acute heart failure syndrome with significant rates of in and out-of-hospital mayor cardiac adverse events (MACE). To evaluate the possible role of neoplastic biomarkers [CA-15.3, CA-19.9 and Carcinoembryonic Antigen (CEA)] as prognostic marker at short- and long-term follow-up in subjects with TTS. Ninety consecutive subjects with TTS were enrolled and followed for a median of 3 years. Circulating levels of CA-15.3, CA-19.9 and CEA were evaluated at admission, after 72 h and at discharge. Incidence of MACE during hospitalization and follow-up were recorded. Forty-three (46%) patients experienced MACE during hospitalization. These patients had increased admission levels of CEA (4.3 ± 6.2 vs. 2.2 ± 1.5 ng/mL, p = 0.03). CEA levels were higher in subjects with in-hospital MACE. At long term follow-up, CEA and CA-19.9 levels were associated with increased risk of death (log rank p < 0.01, HR = 5.3, 95% CI 1.9–14.8, HR = 7.8 95% CI 2.4–25.1, respectively, p < 0.01). At multivariable analysis levels higher than median of CEA, CA-19.9 or both were independent predictors of death at long term (Log-Rank p < 0.01). Having both CEA and CA-19.9 levels above median (> 2 ng/mL, > 8 UI/mL respectively) was associated with an increased risk of mortality of 11.8 (95% CI 2.6–52.5, p = 0.001) at follow up. Increased CEA and CA-19.9 serum levels are associated with higher risk of death at long-term follow up in patients with TTS. CEA serum levels are correlated with in-hospital MACE.

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P1818Descending aortic thoracic diameter: a risk marker for major adverse cardiovascular outcomes in women

European Heart Journal ◽

10.1093/eurheartj/ehz748.0570 ◽

2019 ◽

Vol 40 (Supplement_1) ◽

Author(s):

O L Rueda Ochoa ◽

L R Bons ◽

S Rohde ◽

K E L Ghoud ◽

R Budde ◽

...

Keyword(s):

Heart Failure ◽

Cardiovascular Risk ◽

Cardiovascular Mortality ◽

Total Mortality ◽

Population Based ◽

Cardiovascular Outcomes ◽

Increased Risk ◽

Almost All

Abstract Background Thoracic aortic diameters have been associated with cardiovascular risk factors and atherosclerosis. However, limited evidence regarding the role of thoracic aortic diameters as risk markers for major cardiovascular outcomes among women and men exist. Purpose To evaluate the independent associations between crude and indexed ascending and descending aortic (AA and DA) diameters with major cardiovascular outcomes among women and men and to provide optimal cutoff values associated with increased cardiovascular risk. Methods and results 2178 women and men ≥55 years from the prospective population-based Rotterdam Study underwent multi-detector CT scan of thorax. Crude diameters of the AA and DA were measured and indexed by height, weight, body surface area (BSA) and body mass index (BMI). Incidence of stroke, coronary heart disease (CHD), heart failure (HF), cardiovascular and all-cause mortality were evaluated during 13 years of follow-up. Weight-, BSA-, or BMI-indexed AA diameters showed significant associations with total or cardiovascular mortality in both sexes and height-indexed values showed association with HF in women. Crude AA diameters were associated with stroke in men and HF in women. For DA, crude and almost all indexed diameters showed significant associations with either stroke, HF, cardiovascular or total mortality in women. Only weight-, BSA- and BMI-indexed values were associated with total mortality in men. For crude DA diameter, the risk for stroke increased significantly at the 75th percentile among men while the risks for HF and cardiovascular mortality increased at the 75th and 85th percentiles respectively in women. Conclusions Our study suggests a role for descending thoracic aortic diameter as a marker for increased cardiovascular risk, in particular for stroke, heart failure and cardiovascular mortality among women. The cut points for increased risk for several of cardiovascular outcomes were below the 95th percentile of the distribution of aortic diameters.

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Are Prolonged Ventricular Pauses in Atrial Fibrillation a Marker of Poor Prognosis?

Cardiology ◽

10.1159/000520183 ◽

2021 ◽

Author(s):

Dorte Marie Stavnem ◽

Rakin Hadad ◽

Bjørn Strøier Larsen ◽

Olav Wendelboe Nielsen ◽

Mark Aplin Frederiksen ◽

...

Keyword(s):

Atrial Fibrillation ◽

Poor Prognosis ◽

Pacemaker Implantation ◽

Real Life ◽

Increased Risk ◽

All Cause Mortality ◽

Long Term Prognosis ◽

Rate Limiting

Background: In patients with atrial fibrillation (AF), the long-term prognosis of long electrocardiographic pauses in the ventricular action is not well-studied. Methods: Consecutive Holter recordings in patients with AF (n=200) between 2009-2011 were evaluated, focusing on pauses of at least 2.5 s. Outcomes of interest were all-cause mortality and pacemaker implantation. Results: Forty-three patients (21.5%) had pauses with a mean of 3.2 s and SD of 0.9 s. After a median follow-up of 99 months (ranging 89-111), 47% (20/43) of the patients with, and 45% (70/157) without pauses were deceased. Pauses of ≥ 2.5 s did not constitute a risk of increased mortality: HR = 0.75; (95% CI: 0.34 - 1.66); p = 0.48. Neither did pauses of ≥ 3.0 s: HR = 0.43; (95% CI: 0.06 - 3.20); p = 0.41. Sixteen percent of patients with pauses underwent pacemaker implantation during follow-up. Only pauses in patients referred to Holter due to syncope and/or dizzy spells were associated with an increased risk of pacemaker treatment: HR = 4.7 (95% CI: 1.4-15.9), p = 0.014, adjusted for age, sex and rate-limiting medication. Conclusion: In patients with AF, prolonged electrocardiographic pauses of ≥ 2.5 s or ≥ 3.0 s are not a marker for increased mortality in this real-life clinical study.

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Blood Neutrophil Counts are Associated with Exacerbation Frequency and Mortality in COPD

10.21203/rs.3.rs-16064/v1 ◽

2020 ◽

Author(s):

Mike Lonergan ◽

Alison J Dicker ◽

Megan L Crichton ◽

Holly R Keir ◽

Melissa K. Van Dyke ◽

...

Keyword(s):

Large Population ◽

Real Life ◽

Population Based ◽

Blood Eosinophil ◽

Blood Neutrophil ◽

Disease Heterogeneity ◽

Disease Information ◽

Increased Risk ◽

Eosinophil Counts

Abstract Background Identifying patients with COPD at increased risk of poor outcomes is challenging due to disease heterogeneity. Potential biomarkers need to be readily available in real-life clinical practice. Blood eosinophil counts are widely studied but few studies have examined the prognostic value of blood neutrophil counts (BNC). Methods In a large population-based COPD registry in the East of Scotland (TARDIS: Tayside Allergic and Respiratory Disease Information System), BNC were compared to measures of disease severity and mortality over up to 15 years follow-up. Potential mechanisms of disease modification by BNC were explored in a nested microbiome substudy. Results 178120 neutrophil counts were obtained from 7220 people (mean follow up 9 years) during stable disease periods. Median BNC was 5200cells/µL (IQR 4000-7000cells/µL). Mortality rates among those 34% with elevated BNCs (defined as 6000-15000cells/µL) at the study start were 80% higher (14.0/100 person years v 7.8/100py, P<0.001) than those with BNC in the normal range (2000-6000cells/µL). People with elevated BNC were more likely to be classified as GOLD D (46% v 33% P<0.001), have more exacerbations (mean 2.3 v 1.3/year, P<0.001), and were more likely to have severe exacerbations (13% vs. 5%, P<0.001) in the following year. Eosinophil counts were much less predictive of these outcomes. In a sub-cohort (N=276), patients with elevated BNC had increased relative abundance of Proteobacteria and reduced microbiome diversity. Conclusion High BNC may provide a useful indicator of risk of exacerbations and mortality in COPD patients.

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Supper Timing and Cardiovascular Mortality: The Japan Collaborative Cohort Study

Nutrients ◽

10.3390/nu13103389 ◽

2021 ◽

Vol 13 (10) ◽

pp. 3389

Author(s):

Jingyun Tang ◽

Jia-Yi Dong ◽

Ehab S. Eshak ◽

Renzhe Cui ◽

Kokoro Shirai ◽

...

Keyword(s):

Hemorrhagic Stroke ◽

Proportional Hazards ◽

Cox Proportional Hazards ◽

Stroke Mortality ◽

Hazard Ratios ◽

Risk Of Mortality ◽

Increased Risk ◽

Study Participants

Evidence on the role of supper timing in the development of cardiovascular disease (CVD) is limited. In this study, we examined the associations between supper timing and risks of mortality from stroke, coronary heart disease (CHD), and total CVD. A total of 28,625 males and 43,213 females, aged 40 to 79 years, free from CVD and cancers at baseline were involved in this study. Participants were divided into three groups: the early supper group (before 8:00 p.m.), the irregular supper group (time irregular), and the late supper group (after 8:00 p.m.). Cox proportional hazards regression models were used to calculate hazard ratios (HRs) for stroke, CHD, and total CVD according to the supper time groups. During the 19-year follow-up, we identified 4706 deaths from total CVD. Compared with the early supper group, the multivariable HR of hemorrhagic stroke mortality for the irregular supper group was 1.44 (95% confidence interval [CI]: 1.05–1.97). There was no significant association between supper timing and the risk of mortality from other types of stroke, CHD, and CVD. We found that adopting an irregular supper timing compared with having dinner before 8:00 p.m. was associated with an increased risk of hemorrhagic stroke mortality.

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Role of biomarkers in predicting the occurrence of thyroid neoplasms in radiation-exposed children

Endocrine Related Cancer ◽

10.1530/erc-17-0408 ◽

2018 ◽

Vol 25 (4) ◽

pp. 481-491

Author(s):

Joseph M Shulan ◽

Leonid Vydro ◽

Arthur B Schneider ◽

Dan V Mihailescu

Keyword(s):

Thyroid Cancer ◽

Thyroid Nodules ◽

Subsequent Development ◽

Thyroid Neoplasms ◽

Childhood Cancer Survivors ◽

Malignant Neoplasms ◽

Increased Risk ◽

Need To Evaluate

With increasing numbers of childhood cancer survivors who were treated with radiation, there is a need to evaluate potential biomarkers that could signal an increased risk of developing thyroid cancer. We aimed to examine the relationships between thyrotropin and thyroglobulin levels and the risk of developing thyroid nodules and cancer in a cohort of radiation-exposed children. 764 subjects who were irradiated in the neck area as children were examined and followed for up to 25 years. All subjects underwent a clinical examination, measurements of thyrotropin, thyroglobulin levels and thyroid imaging. At baseline, 216 subjects had thyroid nodules and 548 did not. Of those with nodules, 176 underwent surgery with 55 confirmed thyroid cancers. During the follow-up, 147 subjects developed thyroid nodules including 22 with thyroid cancer. Thyroglobulin levels were higher in subjects with prevalent thyroid nodules (26.1 ng/mL vs 9.37 ng/mL; P < 0.001) and in those who had an initial normal examination but later developed thyroid nodules (11.2 ng/mL vs 8.87 ng/mL; P = 0.017). There was no relationship between baseline thyrotropin levels and the prevalent presence or absence of thyroid nodules, whether a prevalent neoplasm was benign or malignant, subsequent development of thyroid nodules during follow-up or whether an incident nodule was benign or malignant. In conclusion, in radiation-exposed children, higher thyroglobulin levels indicated an increased risk of developing thyroid nodules but did not differentiate between benign and malignant neoplasms. There was no association between the baseline TSH level and the risk of developing thyroid nodules or cancer.

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Risk of benign paroxysmal positional vertigo in patients with depressive disorders: a nationwide population-based cohort study

BMJ Open ◽

10.1136/bmjopen-2018-026936 ◽

2019 ◽

Vol 9 (3) ◽

pp. e026936

Author(s):

Chiao-Lin Hsu ◽

Shih-Jen Tsai ◽

Cheng-Che Shen ◽

Ti Lu ◽

Yao-Min Hung ◽

...

Keyword(s):

Cohort Study ◽

Lupus Erythematosus ◽

Depressive Disorders ◽

Benign Paroxysmal Positional Vertigo ◽

Observation Time ◽

Research Database ◽

Positional Vertigo ◽

Increased Risk ◽

Paroxysmal Positional Vertigo

ObjectiveThe association between depression and benign paroxysmal positional vertigo (BPPV) remains debated. This study aimed to investigate the risk of BPPV in patients with depressive disorders.DesignLongitudinal nationwide cohort study.SettingNational health insurance research database in Taiwan.ParticipantsWe enrolled 10 297 patients diagnosed with depressive disorders between 2000 and 2009 and compared them to 41 188 selected control patients who had never been diagnosed with depressive disorders (at a 1:4 ratio matched by age, sex and index date) in relation to the risk of developing BPPV.MethodsThe follow-up period was defined as the time from the initial diagnosis of depressive disorders to the date of BPPV, censoring or 31 December 2009. Cox proportional hazard regression analysis was used to investigate the risk of BPPV by sex, age and comorbidities, with HRs and 95% CIs.ResultsDuring the 9-year follow-up period, 44 (0.59 per 1000 person-years) patients with depressive disorders and 99 (0.33 per 1000 person-years) control patients were diagnosed with BPPV. The incidence rate ratio of BPPV among both cohorts calculating from events of BPPV per 1000 person-years of observation time was 1.79 (95% CI 1.23 to 2.58, p=0.002). Following adjustments for age, sex and comorbidities, patients with depressive disorders were 1.55 times more likely to develop BPPV (95% CI 1.08 to 2.23, p=0.019) as compared with control patients. In addition, hyperthyroidism (HR=3.75, 95% CI 1.67–8.42, p=0.001) and systemic lupus erythematosus (SLE) (HR=3.47, 95% CI 1.07 to 11.22, p=0.038) were potential risk factors for developing BPPV in patients with depressive disorders.ConclusionsPatients with depressive disorders may have an increased risk of developing BPPV, especially those who have hyperthyroidism and SLE.

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Role of UGT1A1 and ADH gene polymorphisms in pegvisomant-induced liver toxicity in acromegalic patients

Acta Endocrinologica ◽

10.1530/eje-13-0657 ◽

2014 ◽

Vol 170 (2) ◽

pp. 247-254 ◽

Cited By ~ 12

Author(s):

M Filopanti ◽

A M Barbieri ◽

G Mantovani ◽

S Corbetta ◽

V Gasco ◽

...

Keyword(s):

Liver Toxicity ◽

Tertiary Care ◽

Somatostatin Analogs ◽

Liver Function Tests ◽

Regression Analyses ◽

Increased Risk ◽

Adh Gene ◽

Concomitant Medications

ContextHepatotoxicity is one of the most serious adverse effects in acromegalic patients treated with pegvisomant (PEG-V). Recent studies have found an association between this adverse event and the UGT1A1 allele 28 polymorphism associated with Gilbert's syndrome.ObjectiveTo determine whether UGT1A1*28 and alcohol dehydrogenase (ADH) polymorphisms influence liver toxicity during PEG-V treatment.Design and settingMulticenter observational retrospective study conducted in 13 tertiary care endocrinology units in Italy.PatientsA total of 112 patients with active disease resistant to somatostatin analogs (SSTa) and 108 controls were enrolled.InterventionsClinical and biochemical data were recorded by electronic clinical reporting forms. Blood or DNA samples were sent to the coordinating center for genotyping.ResultsNo differences in genotypes between patients and controls were found. During PEG-V therapy liver function tests (LFT), abnormalities and overt hepatotoxicity developed in 17 and 4.5% of patients respectively. Logistic and linear regression analyses showed an association between LFT abnormalities during the follow-up visit and prior events of LFT abnormalities in medical history (odds ratio=1.25;P=0.04) and the number of concomitant medications, other than SSTa (B=3.9;P=0.03). No correlation between LFT alterations and UGT1A1 allele 28 as well as ADH1C and B polymorphisms was found.ConclusionsUGT1A1 allele 28 and ADH1C and B polymorphisms do not predict increased risk of hepatotoxicity during PEG-V therapy. Conversely, patients with multi-therapies and with previous episodes of liver disease should be carefully managed, due to the observed association between these conditions and LFT abnormalities during PEG-V therapy.

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FA05.01: NON-ONCOLOGICAL PULMONARY MORTALITY IS ASSOCIATED WITH OESOPHAGECTOMY FOR CANCER

Diseases of the Esophagus ◽

10.1093/dote/doy089.fa05.01 ◽

2018 ◽

Vol 31 (Supplement_1) ◽

pp. 9-9

Author(s):

Stefan Antonowicz ◽

Sheraz Markar ◽

Tom Wiggins ◽

Hugh Mackenzie ◽

Alan Askari ◽

...

Keyword(s):

Oesophageal Cancer ◽

Conflicts Of Interest ◽

Cancer Recurrence ◽

Disease Diagnosis ◽

Mortality Data ◽

Primary Cancer ◽

Respiratory Mortality ◽

Increased Risk

Abstract Background Improvements in oesophageal cancer care have meant ever more patients are being declared cured. Whether oesophagectomy itself causes long-term non-cancer mortality is not known. This study was conducted to assess the timing and frequency of non-cancer causes of death after oesophagectomy in the UK with a population-based survey. Methods Hospital Episode Statistics provides contemporaneous admission data for all inpatient National Health Service encounters since 2000. A linked database was constructed of all HES encounters with oesophagectomy treatment codes, with Office for National Statistics mortality data, which included cause and date of death. Minimum follow-up was to 5 years. Independent variables potentially predictive of cause of death were entered into logistic regression analyses. Results There were 7204 oesophagectomy patients for which linked mortality data was available. A total of 302 died within 90 days, and a further 5874 died of primary cancer recurrence. Of the remaining 908 non-index-cancer deaths, 238 (26.2%) died of respiratory causes, 210 (23.1%) died of other cancers, 158 (17.4%) died of cardiac diagnoses and 64 (7%) died of cerebrovascular diagnoses. Survival patterns for those dying of cardiac and primary cancer recurrence were similar, with 80% occurring within 2 years of surgery. Non-cancer respiratory mortality was a later occurrence, with 80% occurring by 6 years. A pre-operative pulmonary diagnosis was associated with pulmonary mortality (OR 2.66 95% C.I. 1.49–4.77, P < 0.001), and a pre-operative ischaemic heart disease diagnosis were associated with post-operative cardiac death (OR 2.28 95% C.I. 1.13–4.59, P = 0.021). Long-term respiratory mortality was associated with inpatient respiratory complications in the index encounter (OR 2.60 95% C.I. 1.36 to 4.98, P = 0.004). Comparison to mortality rates after colectomy for cancer revealed 2-fold increased risk of non-cancer pulmonary death after oesophagectomy. Conclusion Oesophagectomy may increase the risk of non-cancer respiratory death in oesophageal cancer survivorship, with implications for peri-operative pathways and follow-up programs. Further work is needed to test whether this is a consequence of reconstruction, peri-operative complications, or progression of pre-morbid diagnoses. Disclosure All authors have declared no conflicts of interest.

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Evaluation of Chronic Transfusion (Tx) Practices in Children with Sickle Cell Disease (SCD): A Survey of STOP II Investigators.

Blood ◽

10.1182/blood.v104.11.3732.3732 ◽

2004 ◽

Vol 104 (11) ◽

pp. 3732-3732 ◽

Cited By ~ 2

Author(s):

Ofelia Alvarez ◽

Scott Miller ◽

Brian Berman ◽

Clark Brown ◽

James Casella ◽

...

Keyword(s):

Iron Overload ◽

Stroke Prevention ◽

Packed Red Blood Cells ◽

Increased Risk ◽

Hb S ◽

Liver Biopsies ◽

Machine Programming ◽

Key Barrier ◽

Total Exchange

The follow-up Stroke Prevention Trial (STOP II) attempts to optimize tx therapy for primary stroke prevention in children with SCD who are at increased risk due to an abnormal Doppler ultrasound. A survey of participating investigators (PIs) was performed in order to assess tx practices; 12 PIs out of 26 responded. To begin chronic tx, 42 % of the PIs preferred erythrocytapheresis (ECP), partial or total exchange to bring hemoglobin (Hb) S <30%, 33% used frequent txs over a month, and the rest used either method. For continuation of chronic tx, 5 PIs used only simple tx for their pts, 2 used partial exchange (phlebotomy and simple tx), and 5 used either simple tx or exchange to transfuse their patients (pts). The most common reason to choose a method was intravenous (IV) accessibility. 4 PIs mentioned that they start with simple tx but later might use exchange if unable to keep Hb S <30% or to avoid iron overload. For simple tx, most PIs ordered 10–15 ml/kg leucoreduced, Rh and Kell compatible, S negative packed red blood cells (PRBC). Pts returned for the next tx, depending on prior pre-tx Hb S (goal < 30%), or every 4 weeks unless Hb S was >30%. Most PIs had a post-tx target hematocrit (Hct) 35–36%, but a third of them did not have one. Pre-tx Hct and/or Hb S (but not post-tx values) were used to predict the timing of the next tx by 83% of the group. When performing ECP, the PIs participated in the decision of how much to exchange pts only 33% of the time; generally ECP was planned by a blood bank physician and/or by machine programming. Chelation began either after 12–30 months of tx (median 18 months), or after serum ferritin 1000–2500 ng/ml (median 2000). 67% of the PIs obtained liver biopsies in all or some of the STOP II pts. Indications for liver biopsies were cited as routine for transfused pts (5 PIs) or depending on ferritin values (6 PIs). Deferoxamine 25–50 mg/kg was infused subcutaneously over 8–10 hours 5–7 nights a week in all pts. Eight PIs reported the use of central venous lines (ports) in some pts to facilitate IV access. Barriers cited to effective chronic tx were: pt compliance with chelation (7 reported it as most important), IV access, pt compliance with tx schedule, hypersplenism, and alloimmunization. We conclude that hematologists :(1) Administer leucoreduced Rh and Kell compatible, S negative PRBC to keep pre-tx Hb S levels <30%, (2) use pre-tx Hb S and Hct to predict next tx, usually every 3–4 weeks, (3) monitor and treat iron overload, and (4) report that poor compliance with chelation is a key barrier to an effective tx program. Although liver biopsy to monitor iron stores and partial/total exchange to limit iron overload are accepted interventions, medical practice still varies.

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