Q1 – 19: Exagamglogene autotemcel (Casgevy®)

!Pre-Infusion iron chelation
Date of last iron chelation therapy dose given pre-infusion will only be enabled if iron chelation therapy was reported on the corresponding Disease Classification (2402) Form.

Questions 1 – 2: Date of last iron chelation therapy dose given pre-infusion

Indicate if the date of the last iron chelation therapy dose administered prior to the start of the preparative regimen is known.
If the date of the last dose is Known, specify the date of the last administration prior to the start of the preparative regimen.

For more information regarding reporting partial or unknown dates, see General Instructions, General Guidelines for Completing Forms.

Questions 3 – 4: Has the recipient received phlebotomy for managing iron overload? (in the 12 months prior to conditioning)

Phlebotomy is a procedure in which blood is removed from the body with the goal of reducing iron overload. Phlebotomy therapy is typically a series of these procedures.

Indicate if phlebotomy was performed in the 12 months prior to the start of the preparative regimen. If Yes, indicate the most recent date of phlebotomy for managing iron overload prior to the start of the preparative regimen.

If phlebotomy was not performed in the 12 months prior to the start of the preparative regimen, or it is not known if performed, select No.

!Complete RBC and Splenomegaly
These questions are applicable to transfusion-dependent thalassemia only, as reported on the Disease Classification (2402) Form.

Questions 5 – 6: Complete red blood cell count (RBC) (most recent prior to collection of infusion product) (transfusion-dependent thalassemia only)

Report the most recent complete red blood cell count (RBC) value and sample collection date obtained prior to collection of the infused product and the collection date.

Report the laboratory value closest to, but before, the product collection. Do not report a value obtained after collection has begun. If multiple values are available prior to collection, report the most recent.

For more information regarding reporting partial or unknown dates, see General Instructions, General Guidelines for Completing Forms.

Questions 7: Did the recipient have a splenomegaly? (transfusion-dependent thalassemia only)

Indicate if the recipient had splenomegaly (i.e., abnormal enlargement of the spleen) at any time since diagnosis. Splenomegaly is often documented during the physician’s physical assessment of the recipient and represents an abnormal finding. Splenomegaly can also be detected by imaging techniques such as ultrasonography, CT or MRI.

If there has been no splenomegaly documented since diagnosis or it is unknown, select No.

!Acute Chest Syndrome
Number of acute chest syndrome (ACS) episodes is applicable to sickle cell disease only, as reported on the Disease Classification (2402) Form, and if acute chest syndrome was reported on the Sickle Cell Pre-Infusion (2030) Form.

Question 8: Number of acute chest syndrome (ACS) episodes (in the 12 months prior to conditioning) (sickle cell disease only)

While the corresponding Sickle Cell Disease (SCD) Pre-Infusion Data (2030) Form collects the total number of episodes in the two years prior to the start of the preparative regimen, this supplemental data form collects the total number of ACS episodes in the 12 months prior to the start of the preparative regimen.

Indicate the total number of ACS episodes in the 12 months prior to the start of the preparative regimen.

If the number of ACS episodes in the 12 months prior to the start of the preparative regimen are not known, leave the question blank and override the error using the code “Unknown.”

!Vaso-occlusive pain, Narcotics / opioids, Non-narcotics analgesics
These questions are applicable to sickle cell disease only, as reported on the Disease Classification (2402) Form, and if vaso-occlusive pain was reported on the Sickle Cell Pre-Infusion (2030) Form.

Question 9: Number of vaso-occlusive pain episodes (in the 12 months prior to conditioning) (sickle cell disease only)

While the corresponding Sickle Cell Disease (SCD) Pre-Infusion Data (2030) Form collects the total number of episodes in the two years prior to the start of the preparative regimen, this supplemental data form collects the total number of ACS episodes in the 12 months prior to the start of the preparative regimen.

Indicate the total number of vaso-occlusive pain episodes in the 12 months prior to the start of the preparative regimen.

If the number of vaso-occlusive pain episodes in the 12 months prior to the start of the preparative regimen are not known, leave the question blank and override the error using the code “Unknown.”

Question 10: Were narcotics / opioids administered? (in the 12 months prior to conditioning) (sickle cell disease only)

While the corresponding Sickle Cell Disease (SCD) Pre-Infusion Data (2030) Form collects if opioids were prescribed for the treatment of pain, this supplemental data form collects if narcotics or opioids were administered for the treatment of pain in the 12 months prior to the start of the preparative regimen.

Indicate if the recipient was administered narcotics or opioids for the treatment of pain in the 12 months prior to the start of the preparative regimen.

If no information is available to determine if narcotics or opioids were administered for treatment of pain in the 12 months prior to the start of the preparative regimen, report Unknown.

Question 11: Were non-narcotic analgesics administered? (in the 12 months prior to conditioning) (sickle cell disease only)

Non-narcotic analgesics may be listed in the medical administration record (MAR) and / or in the progress notes. Examples include, but are not a limited to, acetaminophen and NSAIDS.

Indicate if the recipient was administered non-narcotic analgesics for the treatment of pain in the 12 months prior to the start of the preparative regimen.

If no information is available to determine if non-narcotic analgesics were administered for treatment of pain in the 12 months prior to the start of the preparative regimen, report Unknown.

!CNS Complications
These questions are applicable to transfusion-dependent thalassemia only, as reported on the Disease Classification (2402) Form.

Questions 12 – 13: Was there a central nervous system (CNS) complication? (transfusion-dependent thalassemia only)

A CNS complication is the development of different neurologic signs and symptoms that occur in some recipients with transfusion-dependent thalassemia. CNS complications include the following:

• CNS hemorrhage: bleeding inside the skull
• Encephalopathy (non-infectious): brain inflammation caused by the body’s own immune system attacking healthy brain cells
• Neuropathy: damage or dysfunction of the peripheral motor nerves.
• Seizures: Uncontrolled electrical activity in the brain, which may produce a physical convulsion, minors physical sings, thought disturbances or a combination of symptoms.
• Stroke / transient ischemic attack: interruption of blood supple to parts of the brain resulting in neurologic damage / a brief attack (less than 24 hours) of cerebral dysfunction of vascular origin, with no persistent neurological deficit.

Report Yes if the recipient experienced a CNS complication, for any reason, at any time between diagnosis and the start of the preparative regimen / infusion and select all types. If a CNS event did not occur or no information is available to determine if a CNS event occurred, report No or Unknown, respectively.

!Priapism
Priapism is applicable to sickle cell disease only, as reported on the Disease Classification (2402) Form and if priapism was reported on the Sickle Cell Pre-Infusion (2030) Form.

*Priapism
Questions regarding priapism are applicable to only biologically male recipients.

Question 14: Number of priapism episodes (in the 12 months prior to conditioning) (sickle cell disease only)

While the corresponding Sickle Cell Disease (SCD) Pre-Infusion Data (2030) Form collects priapism episodes at any time between diagnosis and the start of the preparative regimen, this supplemental data form collects the number of priapism episodes in the 12 months prior to the start of the preparative regimen.

If the number of ACS episodes in the 12 months prior to the start of the preparative regimen are not known, leave the question blank and override the error using the code “Unknown.”

!Vascular Impairment / Disorder
Vascular impairment / disorder is applicable to transfusion-dependent thalassemia only, as reported on the Disease Classification (2402) Form.

Question 15: Specify vascular impairment / disorder (transfusion-dependent thalassemia only) (check all that apply)

Deep vein thrombosis (DVT) is a condition characterized by formation of blood clots in deep veins of the body. These clots typically manifest in the lower leg, thigh, or pelvis, but have been noted to occur in the arm.

Pulmonary embolism (PE) is a medical condition where a blood clot is lodged in an artery of the lung, blocking blood flow to that area.

Indicate if the recipient developed a Deep vein thrombosis or Pulmonary embolism between diagnosis and the start of the preparative regimen / infusion. Do not report any DVT or PE that occurred prior to disease diagnosis. If the recipient did not develop either of these vascular impairments, select None.

!Splenic Sequestration
Splenic sequestration is applicable to sickle cell disease only, as reported on the Disease Classification (2402) Form.

Question 16: Number of splenic sequestration events (in the 12 months prior to conditioning) (sickle cell disease only)

While the corresponding Sickle Cell Disease (SCD) Pre-Infusion Data (2030) Form collects the number of splenic sequestration events between diagnosis and the start of the preparative regimen, this supplemental data form collects the number of splenic sequestration events in the 12 months prior to the start of the preparative regimen.

Indicate the total number of splenic sequestration events in the 12 months prior to the start of the preparative regimen.

If the number of splenic sequestration events in the 12 months prior to the start of the preparative regimen are not known, leave the question blank and override the error using the code “Unknown.”

!Mean corpuscular volume (MCV)
Mean corpuscular volume (MCV) is applicable to sickle cell disease only, as reported on the Disease Classification (2402) Form.

Question 17: Mean corpuscular volume (MCV) (most recent prior to collection of infusion product) (sickle cell only)

Specify the recipient’s most recent MCV value prior to collection of the infused product.

Questions 18 – 19: Indirect bilirubin (most recent prior to collection of infusion product)

Indirect (unconjugated) bilirubin serves as a marker for hemolysis or specific hepatic metabolic disorders and provides a baseline for post-infusion comparisons.

Specify if the indirect bilirubin value prior to the collection of the infused product is known. If Known, report the value and units of measurement. If multiple labs are available, use the one closest to the collection start time, even if it is from the same day.

If there is no documentation of the indirect bilirubin level (or the components required to calculate it) prior to product collection, select Unknown.

Section Updates:

Question Number	Date of Change	Add/Remove/Modify	Description	Reasoning (If applicable)
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