Table 2.

Outcomes prioritized by the ASH Guideline Panel on Cardiopulmonary and Kidney Disease

Critical outcomes for decision-making
Q1. 
 • Test accuracy of ECHO to diagnose PH compared with cardiac catheterization 
 • Mortality 
 • Change in patient management 
 • Patient desire to know about abnormal screening 
 • Rate of cardiac catheterization 
 • Anxiety related to abnormal test 
Q2. 
 • Appropriate treatment in appropriate patients 
 • Mortality 
 • Time to diagnosis 
 • Complications related to cardiac catheterization (eg, bleeding, clots, infection, arrhythmia, and pain) 
 • Progression of cardiac dysfunction 
Q3. 
 • Mortality 
 • Quality of life 
 • PH therapy side effects (eg, hypotension, flushing/headache, injection-related pain, liver dysfunction) 
 • Oxygen requirement 
 • Dyspnea, exercise tolerance, 6MWD, and NYHA functional class 
 • Transfusion side effects (eg, alloimmunization, need for and complications related to IV access, iron overload) 
 • Change in pain episodes 
 • Burden of treatment 
 • Cardiac function 
 • Hospitalization rate 
 • Syncope 
Q4. 
 • Rate of cardiac catheterization 
 • Mortality 
 • Change in patient management 
 • Patient desire to know about abnormal screening 
 • Anxiety related to abnormal test 
Q5. 
 • Quality of life 
 • Mortality 
 • Cardiovascular outcomes 
 • Acute and chronic pain rate 
 • Acute chest syndrome rate 
 • Nocturnal enuresis 
 • Priapism 
 • Burden of treatment 
 • Patient desire to know about abnormal screening 
Q6. 
 • End-stage renal disease 
 • Mortality 
 • Quality of life/function 
 • Burden of treatment 
 • Worsening proteinuria 
 • Hospitalization rate 
 • Hyperkalemia 
 • Hypotension 
 • Acute kidney injury 
Q7. 
 • Overall mortality 
 • Transplant organ survival 
 • Quality of life 
 • Transplant-related mortality 
 • Treatment burden related to transplant vs dialysis 
 • Improvement in anemia 
 • Hospitalization 
 • Pain episodes 
Q8. 
 • Mortality 
 • Quality of life 
 • Improvement in renal function 
 • Improvement in hemoglobin 
 • Acute and chronic pain rate 
 • Burden of treatment 
 • Bone marrow suppression 
 • Hypertension 
 • Thrombosis 
Q9. 
 • Mortality 
 • Stroke 
 • Cardiovascular events 
 • Worsening renal function 
 • Quality of life/function 
 • Hypotension 
 • Burden of treatment (eg, emergency department visits, adherence, monitoring) 
 • Medication side effects 
 • Hospitalization rate 
Q10. 
 • Major bleeding 
 • Risk of recurrent pulmonary embolism 
 • Risk of recurrent deep vein thrombosis 
 • Quality of life 
 • Mortality 
 • SCD-related complications 
 • Treatment burden 
 • Postthrombotic syndrome 
Critical outcomes for decision-making
Q1. 
 • Test accuracy of ECHO to diagnose PH compared with cardiac catheterization 
 • Mortality 
 • Change in patient management 
 • Patient desire to know about abnormal screening 
 • Rate of cardiac catheterization 
 • Anxiety related to abnormal test 
Q2. 
 • Appropriate treatment in appropriate patients 
 • Mortality 
 • Time to diagnosis 
 • Complications related to cardiac catheterization (eg, bleeding, clots, infection, arrhythmia, and pain) 
 • Progression of cardiac dysfunction 
Q3. 
 • Mortality 
 • Quality of life 
 • PH therapy side effects (eg, hypotension, flushing/headache, injection-related pain, liver dysfunction) 
 • Oxygen requirement 
 • Dyspnea, exercise tolerance, 6MWD, and NYHA functional class 
 • Transfusion side effects (eg, alloimmunization, need for and complications related to IV access, iron overload) 
 • Change in pain episodes 
 • Burden of treatment 
 • Cardiac function 
 • Hospitalization rate 
 • Syncope 
Q4. 
 • Rate of cardiac catheterization 
 • Mortality 
 • Change in patient management 
 • Patient desire to know about abnormal screening 
 • Anxiety related to abnormal test 
Q5. 
 • Quality of life 
 • Mortality 
 • Cardiovascular outcomes 
 • Acute and chronic pain rate 
 • Acute chest syndrome rate 
 • Nocturnal enuresis 
 • Priapism 
 • Burden of treatment 
 • Patient desire to know about abnormal screening 
Q6. 
 • End-stage renal disease 
 • Mortality 
 • Quality of life/function 
 • Burden of treatment 
 • Worsening proteinuria 
 • Hospitalization rate 
 • Hyperkalemia 
 • Hypotension 
 • Acute kidney injury 
Q7. 
 • Overall mortality 
 • Transplant organ survival 
 • Quality of life 
 • Transplant-related mortality 
 • Treatment burden related to transplant vs dialysis 
 • Improvement in anemia 
 • Hospitalization 
 • Pain episodes 
Q8. 
 • Mortality 
 • Quality of life 
 • Improvement in renal function 
 • Improvement in hemoglobin 
 • Acute and chronic pain rate 
 • Burden of treatment 
 • Bone marrow suppression 
 • Hypertension 
 • Thrombosis 
Q9. 
 • Mortality 
 • Stroke 
 • Cardiovascular events 
 • Worsening renal function 
 • Quality of life/function 
 • Hypotension 
 • Burden of treatment (eg, emergency department visits, adherence, monitoring) 
 • Medication side effects 
 • Hospitalization rate 
Q10. 
 • Major bleeding 
 • Risk of recurrent pulmonary embolism 
 • Risk of recurrent deep vein thrombosis 
 • Quality of life 
 • Mortality 
 • SCD-related complications 
 • Treatment burden 
 • Postthrombotic syndrome 

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