NIH-NHLBI CureSCi CDEs (version 1.0)
| Domain . | Subdomain . | Class . | Recommendations . | ||
|---|---|---|---|---|---|
| Participant characteristics | Demographics | C | Demographics | ||
| General health history | C | Baseline abnormal hematopoiesis | Behavioral history short form | ||
| Transfusion history | Medical history | Surgical history | |||
| S | Behavioral history | Medical history supplemental elements | |||
| Hospitalization form | Sleep assessment (ped form) | ||||
| Social history | C | Social status | |||
| S | Education school questionnaire | Social determinants screen | |||
| ACEs screen children (1-17 years) | ACEs screen adults ( ≥18 years) | ||||
| Acute anemia | Chronic anemia | ||||
| Disease and treatment-related events | Asthma | S | Asthma outcomes instrument recommendations (highly recommended) | ||
| Asthma outcomes | Over-read spirometry report form | ||||
| Fertility/bone | C | Endocrine, infertility, and bone health | |||
| Lung | C | 6-minute walk test | Pulmonary function test | ||
| Lung disease assessments guidelines | |||||
| S | PROMIS dyspnea functional limitations | ||||
| Pulmonary hypertension | PROMIS dyspnea severity | ||||
| Pain | C | Acute chest syndrome | SCD-related acute painful episodes | ||
| Priapism | C | Priapism core | |||
| S | Priapism Impact Profile (PIP) | Priapism questionnaire | |||
| Renal | C | Renal function assessments | |||
| Spleen | C | Acute spleen | Chronic spleen | ||
| S | Spleen assessment from the pediatric HU phase 3 clinical trial (BABY HUG) | ||||
| Other | S | Leg ulcers | Retinopathy | Avascular necrosis | |
| Chronic malnutrition | Guidelines malnutrition identification | ||||
| Assessments and examinations | Imaging diagnostics | C | Cardiac MRI | Echocardiogram | Brain MRI |
| S | Functional MRI | Brain MRA | Imaging TCD | ||
| Laboratory tests | C | Genetic diagnostic testing | Hemoglobin variant analysis | ||
| S | Immune function form | Lab assessments-genetics/assays | |||
| Nonimaging | S | Electrocardiogram | |||
| Physical exam | S | Physical exam | NIH Stroke Scale | ||
| Vital signs | C | Vital signs and blood gases | |||
| Treatment and interventions | Drugs | C | Prior and concomitant medications | ||
| S | PROMIS Medical Adherence Scale (PMAS) | Asthma medications list | |||
| Therapies | C | Drug product | Hematopoietic cellular transplant infusion | ||
| Genetics and assays summary of recommendations | |||||
| S | Adhesion and viscosity | Apheresis | Conditioning regimen | ||
| E | Adhesion molecules assay | ||||
| Adverse events and toxicities | C | Cytopenia | Genotoxicity | Iron overload | |
| Infusion-related toxicity | Infection form | ||||
| S | Adverse events | New malignancy | Toxicity form | ||
| Cellular therapy essential data follow-up form | |||||
| Outcomes and end points | PROs | C | See details in Table 1 | ||
| Mortality | C | Death form | |||
| Domain . | Subdomain . | Class . | Recommendations . | ||
|---|---|---|---|---|---|
| Participant characteristics | Demographics | C | Demographics | ||
| General health history | C | Baseline abnormal hematopoiesis | Behavioral history short form | ||
| Transfusion history | Medical history | Surgical history | |||
| S | Behavioral history | Medical history supplemental elements | |||
| Hospitalization form | Sleep assessment (ped form) | ||||
| Social history | C | Social status | |||
| S | Education school questionnaire | Social determinants screen | |||
| ACEs screen children (1-17 years) | ACEs screen adults ( ≥18 years) | ||||
| Acute anemia | Chronic anemia | ||||
| Disease and treatment-related events | Asthma | S | Asthma outcomes instrument recommendations (highly recommended) | ||
| Asthma outcomes | Over-read spirometry report form | ||||
| Fertility/bone | C | Endocrine, infertility, and bone health | |||
| Lung | C | 6-minute walk test | Pulmonary function test | ||
| Lung disease assessments guidelines | |||||
| S | PROMIS dyspnea functional limitations | ||||
| Pulmonary hypertension | PROMIS dyspnea severity | ||||
| Pain | C | Acute chest syndrome | SCD-related acute painful episodes | ||
| Priapism | C | Priapism core | |||
| S | Priapism Impact Profile (PIP) | Priapism questionnaire | |||
| Renal | C | Renal function assessments | |||
| Spleen | C | Acute spleen | Chronic spleen | ||
| S | Spleen assessment from the pediatric HU phase 3 clinical trial (BABY HUG) | ||||
| Other | S | Leg ulcers | Retinopathy | Avascular necrosis | |
| Chronic malnutrition | Guidelines malnutrition identification | ||||
| Assessments and examinations | Imaging diagnostics | C | Cardiac MRI | Echocardiogram | Brain MRI |
| S | Functional MRI | Brain MRA | Imaging TCD | ||
| Laboratory tests | C | Genetic diagnostic testing | Hemoglobin variant analysis | ||
| S | Immune function form | Lab assessments-genetics/assays | |||
| Nonimaging | S | Electrocardiogram | |||
| Physical exam | S | Physical exam | NIH Stroke Scale | ||
| Vital signs | C | Vital signs and blood gases | |||
| Treatment and interventions | Drugs | C | Prior and concomitant medications | ||
| S | PROMIS Medical Adherence Scale (PMAS) | Asthma medications list | |||
| Therapies | C | Drug product | Hematopoietic cellular transplant infusion | ||
| Genetics and assays summary of recommendations | |||||
| S | Adhesion and viscosity | Apheresis | Conditioning regimen | ||
| E | Adhesion molecules assay | ||||
| Adverse events and toxicities | C | Cytopenia | Genotoxicity | Iron overload | |
| Infusion-related toxicity | Infection form | ||||
| S | Adverse events | New malignancy | Toxicity form | ||
| Cellular therapy essential data follow-up form | |||||
| Outcomes and end points | PROs | C | See details in Table 1 | ||
| Mortality | C | Death form | |||
ACEs: adverse childhood experiences; Class: classification; C: core; E: exploratory; HU: hydroxyurea; MRA: magnetic resonance angiography; MRI: magnetic resonance imaging; Ped: pediatric; S: supplemental; TCD: transcranial Doppler.
Publicly available at https://curesickle.org/system/files/Sickle_Cell_Disease_CDE_Highlight_Summary.pdf.