Questions 451 – 453: Specify the hemoglobinopathy classification

Indicate the hemoglobinopathy classification of the primary disease for infusion.

Sickle cell disease: A group of disorders that adversely affect the body’s production of hemoglobin, the component in red blood cells that delivers oxygen throughout the body. Individuals with these disorders possess atypical hemoglobin molecules, called hemoglobin “S,” which can distort the red blood cell morphology into a sickle, or crescent, shape.

Transfusion dependent thalassemia: Previously described as “beta thalassemia major” on CIBMTR forms. Transfusion-dependent thalassemia is a blood disorder that reduces the production of hemoglobin in the body and is defined as requiring eight or more transfusion events per year for two years or more for treatment of symptomatic anemia.

If Transfusion dependent thalassemia is selected, specify the transfusion dependent thalassemia as either Transfusion dependent beta thalassemia (known as “beta thalassemia major” on prior CIBMTR forms) or Other transfusion dependent thalassemia in question 452.

Other hemoglobinopathy: If the recipient is diagnosed with a hemoglobinopathy other than sickle cell disease or transfusion dependent thalassemia, select Other hemoglobinopathy and specify the type in question 453.

Question 454: Did the recipient receive gene therapy to treat the hemoglobinopathy?

Indicate if the recipient received a gene therapy to treat the hemoglobinopathy. If the recipient received a gene therapy, indicate Yes. The Cellular Therapy Product (4003) Form and Cellular Therapy Infusion (4006) Form will also come due to collect more information regarding this gene therapy.

Questions 455 – 457: Was tricuspid regurgitant jet velocity (TRJV) measured by echocardiography?

Tricuspid regurgitant jet velocity (TRJV) measurements are used in determining the pulmonary artery pressure for patients with hemolytic disorders. An elevated TRJV is an indication of pulmonary hypertension, a condition common in adults with hemolytic diseases. TRJV can be determined by echocardiography (ECHO); this information is typically documented in the echocardiogram report.

Report whether the TRJV was measured by echocardiography prior to the start of the preparative regimen / infusion. Seek physician clarification, as needed, if the results are unclear.

If the TRJV was measured by echocardiography, select Yes and indicate if the TRJV value is Known or Unknown in question 456. If Known, specify the TRJV value documented in the laboratory report in question 457. If the TRJV was measure multiple times prior to the start of the preparative regimen / infusion by an echocardiography, report the most recent value.

If the TRJV was not measured or it is not known if measured by echocardiography at the diagnosis of transfusion dependent thalassemia, report No or Unknown, respectively and continue with question 458.

Questions 458 – 460: Was liver iron content (LIC) tested within 6 months prior to infusion?

Transfusion support for hemolytic diseases can often lead to iron build up or accumulation in the liver and other target organs. Liver iron content (LIC) is commonly used to measure total iron storage for recipients at risk of hemosiderosis. LIC is a more sensitive method of testing for measuring the level of iron in the liver. Common methods include, but are not limited to, liver biopsy, T2*MRI, and FerriScan.

Report Yes if the liver iron content was assessed within six months prior to the start of the preparative regimen / infusion. Report the value and unit of measure documented in the laboratory report in question 459. Also report the method used to estimate the LIC in question 460. If the method of assessment is not listed, select Other.

Report No if liver iron content was not assessed or it is not known if assessed within six months prior to the start of the preparative regimen / infusion and continue with question 461.

Questions 461 – 462: Is the recipient red blood cell transfusion dependent?

Indicate if the recipient is red blood cell transfusion dependent at any time prior to the start of the preparative regimen / infusion. In this context, “red blood cell transfusion dependent” is defined as requiring transfusions to maintain hemoglobin 9 – 10 d / dL.

If the recipient the recipient was red blood cell transfusion dependent at any time prior to the start of the preparative regimen / infusion, report Yes and specify the year of the first transfusion (since diagnosis) in question 462.

If the recipient was never red blood cell transfusion dependent at any time prior to the start of the preparative regimen / infusion, report No and continue with question 469.

Question 463: Was iron chelation therapy given at any time since diagnosis?

Iron chelation therapy is commonly used for recipients to prevent or reduce iron overload. Iron chelation therapy is the removal of excess iron from the body using drugs such as Deferrioxamine (Desferal) or Deferasirox (Jadenu, Exjade).

Select Yes if iron chelation therapy was given at any time since diagnosis. If iron chelation was not given or it is unknown whether iron chelation therapy was given, select No or Unknown, respectively and continue with question 469.

Question 464: Did iron chelation therapy meet the following criteria: initiated within 18 months of the first transfusion and administered for at least 5 days / week (either oral or parenteral iron chelation medication)

Indicate if the iron chelation therapy was initiated within 18 months of the first transfusion and administered for at least five days a week (either oral or parenteral iron chelation medication).

If iron chelation therapy met the criteria listed above, report Yes, iron chelation therapy given as specified and continue with question 467.

If iron chelation therapy was given but does not meet the specified criteria, report No, iron chelation therapy given, but does not meet criteria and continue with question 465.

If iron chelation therapy was given but administration details are unavailable, report Iron chelation therapy given, but details of administration unknown and continue with question 467.

Questions 465 – 466: Specify reason criteria not met

If iron chelation therapy was given but does not meet the criteria specified in question 464, indicate why the criteria was not met. If the reason is not listed, report Other and specify the reason criteria were not met.

Questions 467 – 468: Year iron chelation therapy started

Indicate if the year iron chelation therapy was started is Known or Unknown. If Known, specify the year when iron chelation therapy began. If the start date of iron chelation therapy is not known, report Unknown and continue with question 469.

Questions 469 – 470: Did the recipient have hepatomegaly? (> 2 cm below costal margin):

Indicate if the recipient had hepatomegaly (i.e. abnormal enlargement of the liver) at the last evaluation prior to the start of the preparative regimen / infusion. Hepatomegaly is often documented during the physician’s physical assessment of the recipient and represents an abnormal finding. If Yes, report the liver measurement (in centimeters below the right costal margin).

If hepatomegaly was not present or is not known if it was present or absent at the last evaluation prior to the start of the preparative regimen, indicate No or Unknown and continue with question 471.

Questions 471 – 473: Was a liver biopsy performed at any time since diagnosis?

Evaluation of liver tissue may be necessary to determine the extent of the recipient’s disease. Indicate if a liver biopsy was performed at any time since diagnosis.

If Yes, report if the assessment date was Known or Unknown in question 472. If Known, report the date of the liver biopsy in question 473. If multiple liver biopsies were performed since diagnosis, report the date of the most recent biopsy.

If the date of the liver biopsy is partially known, use the process described for reporting partial or unknown dates in General Instructions, Guidelines for Completing Forms; check the box Date estimated.

If a liver biopsy was not performed or it is unknown it a biopsy was performed at any time since diagnosis, report No and continue with question 479.

Questions 474: Was there evidence of liver cirrhosis?

Indicate if the liver biopsy performed on the date reported above (in question 473) showed evidence of liver cirrhosis. Select Yes if the biopsy showed characteristics of liver cirrhosis were present.

Report No if the biopsy reported did not show characteristics of liver cirrhosis. If the results are unclear, seek physician clarification.

Select Unknown if no information is available to determine if the biopsy showed characteristics of liver cirrhosis.

Questions 475 – 476: Was there evidence of liver fibrosis?

Indicate if the liver biopsy performed on the date reported above (in question 473) showed evidence of liver fibrosis. Select Yes if the biopsy showed characteristics of liver fibrosis and specify the type of fibrosis observed.

  • Bridging: Bands of fibrous tissue and collagen which span portal spaces and/or centrilobular spaces creating a “bridge-like” appearance
  • Periportal: Fibrous expansion of portal fields with fibrosis extending along the terminal portal veins
  • Other: Select this option if the type of fibrosis present is not listed.
  • Unknown: Select this option if it if known fibrosis was present but the type is not known.

Report No if the biopsy did not show characteristics of liver fibrosis. If the results are unclear, seek physician clarification.

Select Unknown if no information is available to determine if the biopsy showed characteristics of liver fibrosis.

Questions 477: Was there evidence of chronic hepatitis?

Indicate if the liver biopsy performed on the date reported above (in question 471) showed evidence of chronic hepatitis. Select Yes if the biopsy showed characteristics of chronic hepatitis. Report No if the biopsy reported did not show characteristics of chronic hepatitis. If the results are unclear, seek physician clarification.

Select Unknown if no information is available to determine if the biopsy showed characteristics of chronic hepatitis or if the results were inconclusive.

Question 478: Was documentation submitted to the CIBMTR? (e.g., liver biopsy)

Indicate whether a liver biopsy is attached to support / clarify the center’s responses to questions 471 – 477. For further instructions on how to attach documents in FormsNet3SM, refer to the Training Guide.

Question 479: Is there evidence of abnormal cardiac iron deposition based on an MRI of the heart at time of infusion?

A cardiac MRI may be performed to assess cardiac iron deposition. This information is typically listed within the MRI interpretation of the report.

Indicate Yes if cardiac MRI shows evidence of abnormal cardiac iron deposition at the time of infusion. Select No if there is no evidence of abnormal cardiac iron deposition at the time of infusion. If multiple MRIs were performed, report the results based on the most recent scan prior to the start of the preparative regimen / infusion.

Question 480: Did the recipient have a splenectomy at any time prior to infusion?

Indicate Yes or No if the recipient had a splenectomy at any time prior to the start of the preparative regimen / infusion. If it not known whether a splenectomy was performed at any time prior to the start of the preparative regimen / infusion, report Unknown.

Questions 481 – 482: Serum iron

A serum iron test is used to determine how much iron is in the serum. If the serum iron level is lower than normal, it indicates the body’s iron stores are low (iron deficiency). If the serum iron level is higher than normal, it could indicate hemochromatosis, a condition that causes the body to store too much iron.

Indicate whether the serum iron was Known or Unknown at the last evaluation prior to the start of the preparative regimen / infusion. If Known, report the value and unit of measure documented on the laboratory report in question 482. If Unknown, continue with question 483.

Questions 483 – 484: Total iron binding capacity (TIBC)

Total iron binding capacity (TIBC) is a test used to gauge the total amount of iron in the blood.

Indicate whether the TIBC was Known or Unknown at the last evaluation prior to the start of the preparative regimen / infusion. If Known, report the value and unit of measure documented on the laboratory report in question 484. If Unknown, continue with question 485.

Questions 485 – 486: Total Serum bilirubin

Indicate whether the total serum bilirubin was Known or Unknown at the last evaluation prior to the start of the preparative regimen / infusion. If Known, report the value and unit of measure documented on the laboratory report in question 486. If Unknown, continue with question 487.

Question 487: Upper limit of normal for total serum bilirubin:

Indicate the upper limit of normal for the total serum bilirubin documented on the laboratory report.

Section Updates:

Question Number Date of Change Add/Remove/Modify Description Reasoning (If applicable)
. . . . .
Last modified: Dec 22, 2020

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