Q1 – 34: Exagamglogene autotemcel (Casgevy®)

Question 1: Did the recipient experience any infection event?

The intent of this question is to capture any and all infections, regardless of whether medical intervention was initiated.

Report any documented infection occurring in the current reporting period, even if the recipient remained untreated or if the infection did not meet the “clinically significant” threshold used on other CIBMTR forms (i.e., Post-Infusion Follow-Up (2100) Form).

If the infection occurring in the current reporting period was treated and reported on the corresponding follow-up form, such as the Post-Infusion Follow-Up (2100) Form, re-report the infection by selecting Yes.

Question 2: Did the recipient experience a grade 3 or higher bleeding event?

A grade 3 or higher bleeding event is defined as a severe or medically significant but not immediately life threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care ADL or severe impact on age-appropriate normal daily activity (pediatric).

For additional details on the criteria used for grading adverse events refer to CTCAE v6.

Specify if the recipient experienced a grade 3 or higher bleeding event in the current reporting period. If the grade 3 or higher bleeding event did not occur in the current reporting period or is unknown, select No.

*Reporting Multiple Bleeding Events
Complete Specify the bleeding event through Was the recipient hospitalized (or in hospital) for this event questions to report all grade 3 or higher bleeding events occurring in the current reporting period by adding an additional instance in FormsNet3^SM.

Questions 3 – 6: Specify the bleeding event

Indicate the grade 3 or higher bleeding event that occurred in the current reporting period, using the table below, and specify the date of the bleeding event. For more information regarding reporting partial or unknown dates, see General Instructions, General Guidelines for Completing Forms.

If a bleeding event other than those listed occurred in the current reporting period, select Other bleeding event and specify.

Table 1. Bleeding Events and Grading Criteria

Event – Definition	Grade 3	Grade 4	Grade 5
Epistaxis: A disorder characterized by bleeding from the nose	Transfusion; invasive intervention indicated (e.g., hemostasis of bleeding site)	Life-threatening consequences; urgent intervention indicated	Death
Hematuria: A disorder characterized by laboratory test results that indicate blood in the urine	Gross hematuria; transfusion, IV medications, or hospitalization indicated; elective invasive intervention indicated; limiting self-care ADL or severe impact on age appropriate normal daily activity (pediatric)	Life-threatening consequences; urgent invasive intervention indicated	Death
Intercranial hemorrhage: A disorder characterized by bleeding from within the cranium.	Ventriculostomy, ICP monitoring, intraventricular thrombolysis, or invasive intervention indicated; hospitalization	Life-threatening consequences; urgent invasive intervention indicated	Death
Lower GI hemorrhage or rectal bleeding: A disorder characterized by bleeding from the lower GI or rectum.	Hospitalization	Life-threatening consequences; urgent invasive intervention indicated	Death
Menorrhagia: A disorder characterized by abnormally heavy vaginal bleeding during menses	Hospitalization; operative intervention indicated (e.g., hysterectomy)	Life-threatening consequences; urgent invasive intervention indicated	Death
Upper GI hemorrhage: A disorder characterized by bleeding from the upper gastrointestinal tract (oral cavity, pharynx, esophagus, and stomach)	Hospitalization	Life-threatening consequences; urgent invasive intervention indicated	Death

Question 7: Was the recipient hospitalized (or in a hospital) for this event?

For the grade 3 or higher bleeding event reported in this instance, indicate if the recipient hospitalized for the event or was the recipient in the hospital at the time of bleeding event.

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

Question 8: Did the recipient have hepatomegaly? (sickle cell disease only)

Indicate if the recipient had hepatomegaly (i.e., abnormal enlargement of the liver) in the current reporting period. Hepatomegaly may be documented during the physician’s physical assessment of the recipient and represents an abnormal finding or detected through imaging.

If the recipient does not have hepatomegaly or it not known / not documented, report No.

!Complete RBC
Complete RBC is applicable to transfusion-dependent thalassemia only, as reported on the Disease Classification (2402) Form.

Questions 9 – 10: Complete red blood cell count (RBC) (transfusion-dependent thalassemia only)

Report the most recent complete red blood cell count (RBC) value and sample collection date obtained in this reporting period.

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

!Splenomegaly and Non-narcotic analgesics
These questions are applicable to sickle cell disease only, as reported on the Disease Classification (2402) Form.

Question 11: Did the recipient have a splenomegaly? (sickle cell disease only)

Indicate if the recipient had splenomegaly (i.e., abnormal enlargement of the spleen) in this reporting period. 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 it is unknown if the recipient had splenomegaly in the current reporting period, select No.

Question 12: Were non-narcotic analgesics administered? (sickle cell disease only)

Non-narcotic analgesics may be listed in the medical administration record (MAR) and / or in the progress notes. Examples include acetaminophen and NSAIDs.

Indicate whether the recipient was administered on-narcotic analgesics during the current reporting period. If it is not known if non-narcotic analgesics were administered in the current reporting period, select Unknown.

!Hemoglobin level
This questions does not apply to the 100 day follow-form form and will be disable. The question will be enabled starting on the 6 month follow-up form.

Question 13: Did the recipient experience a hemoglobin level <8 g/dL lasting for more than three months? (occurring after Day +100)

Hemoglobin is a molecule in red blood cells that delivers oxygen to tissues throughout the body. A low hemoglobin count is considered “anemia” and blood transfusions, or growth factors may be required to increase the hemoglobin level.

*Chronic anemia
If chronic anemia is documented in the reporting, select Yes regardless of anything ese.

Check only one response:

• Yes:
  • Within a 3 month time period, the hemoglobin was < 8 g/dL on two different days, regardless of receiving transfusions
  • If the recipient is getting regular transfusions in this reporting period to maintain >8 g/dL (documented in the blood order/transfusion order).
  • Chronic anemia: see blue note box above

• No:
  • The hemoglobin level has never been < 8 g/dL in this reporting period
  • The hemoglobin level has been < 8 g/dL for less than 3 months, regardless of receiving transfusions.

• Previously reported: The recipient’s hemoglobin levels were < 8 g/dL lasting for more than three months in the previous reporting period, and it persists into this reporting period.

• Unknown: There were no CBCs obtained or there no information about transfusions in this reporting period. This option should be rarely used.

Questions 14 – 17: Did autoimmune hemolytic anemia develop or persist?

Autoimmune hemolytic anemia refers to the destruction (hemolysis) of red blood cells by the recipient’s own immune system, resulting in low hemoglobin/anemia. Laboratory studies are the most common method of disease detection, usually involving a complete blood cell count and peripheral blood smear.

Indicate if autoimmune hemolytic anemia developed in the current reporting period. Do not report autoimmune hemolytic anemia that was diagnosed pre-infusion and persisted post-infusion. If Yes, specify the onset date and the status at the end of the reporting period. Resolved means resolution of anemia with completion of therapy directed at the autoimmune hemolysis or as document by physician.

If autoimmune hemolytic anemia persisted from the previous reporting period into the current reporting period, report Yes it developed or persisted, Yes, the onset date was previously reported and the status at the end of the reporting period. The Yes option for the previously reported question is only applicable for the six-month and annual reporting periods.

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

Questions 18 – 21: Did aplastic anemia develop or persist?

Aplastic anemia is a hematologic condition defined by peripheral blood cytopenia(s) and markedly hypocellular marrow with pancytopenia and the bone marrow does not produce enough red blood cells, white blood cells, or platelets for the body.

Indicate if aplastic anemia developed in the current period. Do not report aplastic anemia that was diagnosed pre-infusion and persisted post-infusion. If Yes, specify the onset date and the status at the end of the reporting period. Resolved means normalization of peripheral blood cytopenia and normal cellular marrow or as document by physician.

If aplastic anemia persisted from the previous reporting period into the current reporting period, report Yes it developed or persisted, Yes, the onset date was previously reported, and the status at the end of the reporting period. The Yes option for the previously reported question is only applicable for the six-month and annual reporting periods.

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

Questions 22 – 25: Did immune thrombocytopenia develop or persist?

Immune thrombocytopenia is the destruction of platelets by the immune system. This is typically a clinical diagnosis and platelet specific antibodies are not routinely ordered due to their low sensitivity and specificity. However, if platelet specific antibodies were tested for and found to be present this would support a diagnosis of immune thrombocytopenia. A clinical diagnosis should be confirmed if the provider notes are unclear.

Indicate if immune thrombocytopenia developed in the current period. Do not report immune thrombocytopenia that was diagnosed pre-infusion and persisted post-infusion. If Yes, specify the onset date and the status at the end of the reporting period. Resolved means resolution of thrombocytopenia with completion of therapy directed at the immune mediated low platelets or as document by physician.

If immune thrombocytopenia persisted from the previous reporting period into the current reporting period, report Yes it developed or persisted, Yes, the onset date was previously reported, and the status at the end of the reporting period. The Yes option for the previously reported question is only applicable for the six-month and annual reporting periods.

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

Questions 26 – 30: Did another new non-malignant hematologic disorder develop or persist?

Look for the original list from Vertex: include but are not limited to, autoimmune neutropenia, Evan syndrome,

Indicate if another new non-malignant hematologic disorder developed in the current period. If Yes, specify the onset date and the status at the end of the reporting period. Resolved means as document by physician.

If another new non-malignant hematologic disorder persisted from the previous reporting period into the current reporting period, report Yes it developed or persisted, Yes, the onset date was previously reported, and the status at the end of the reporting period. The Yes option for the previously reported question is only applicable for the six-month and annual reporting periods.

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

Questions 31 – 34: Did the recipient develop a solid tumor new malignancy? (also complete Subsequent Neoplasms Form 3500)

Indicate whether the recipient was diagnosed with a new solid tumor malignancy in the current reporting period. Do not report recurrence, progression, or transformation of the recipient’s primary disease (disease for which the infusion was performed), or relapse of a prior solid tumor malignancy.

If a new solid tumor malignancy was diagnosed during the reporting period, report Yes. The new solid tumor malignancy will also need to be reported on the corresponding Post-Infusion Follow-Up (2100) Form and a Subsequent Neoplasm (3500) Form should be completed. In addition, specify the solid tumor – T, solid tumor – N, and solid tumor – M.

Section Updates:

Question Number	Date of Change	Add/Remove/Modify	Description	Reasoning (If applicable)
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CIBMTR® (Center for International Blood and Marrow Transplant Research) is a research collaboration between the Medical College of Wisconsin and NMDP.