This section collects the data known as “best response to transplant.” The purpose of this section is to report the recipient’s best response to the planned course of the HCT. This includes response to any therapy given for post-HCT maintenance or consolidation, and does not include response to treatment given for relapsed, progressive or persistent disease. Best response is often achieved in the first 100 days. However, for some diseases such as multiple myeloma and CLL, the best response to HCT may take longer.
If the recipient relapses / progresses post-HCT and receives therapy for the disease relapse/progression, the response to that additional therapy should not be reported in this section. The best response prior to the relapse / progression should be reported. Reporting periods subsequent to that in which best response prior to the start of unplanned was reported will indicate that best response was previously reported.
Tandem Transplants: For recipients receiving a tandem transplant, the best response to the prior transplant (i.e., HCT #1 of the tandem) depends on the pre-transplant disease status.
- If the recipient was in complete remission at the time of HCT #1, report the best response to transplant as “Continued complete remission (CCR)”.
- If the recipient was not in complete remission at the time of HCT #1, and no disease assessments (including labs and / or physician’s exams) occurred in the reporting period, between HCT #1 and HCT #2 of their tandem transplant, report Not evaluated. However, ensure the best response to transplant and the current disease status are reported consistently
- If the recipient was not in complete remission at the time of HCT #1, and achieved complete remission prior to HCT #2 of their tandem transplant, report Complete remission, the date which complete remission was achieved
- If the recipient was not in complete remission at the time of HCT #1 and did not achieve complete remission in response to HCT #1 and prior to HCT #2 of their tandem transplant, report Not in complete remission (NCR)
Question 75: Compared to the disease status prior to the preparative regimen, what was the best response to HCT since the date of the last report? (Include response to any therapy given for post-HCT maintenance or consolidation, but exclude any therapy given for relapsed, persistent, or progressive disease)
If the recipient was already in CR at the start of the preparative regimen, check Continued complete remission (CCR).
If the recipient achieved CR post-HCT (excluding unplanned therapy), check Complete remission (CR).
If the recipient has not achieved a post-HCT CR to date, check Not in complete remission.
If the recipient’s disease status was not evaluated post-HCT, check Not evaluated. This option is not commonly used, as this would indicate that no tests (radiological, laboratory, or a clinical assessment) were performed to assess the CR status at any time during the reporting period.
If the recipient never achieved a post-transplant complete response and started unplanned therapy, given for relapsed, persistent, or progressive disease, in a previous reporting period, indicate “not evaluated.”
Example 1: A recipient with neuroblastoma is not in complete remission prior to transplant, in the 100-day reporting period the recipient receives a tandem transplant. Between HCT 1 and HCT 2 the only disease assessment performed was a clinical evaluation. In this case either option would be appropriate to answer for the best response: “Not evaluated” or “Not in complete remission (NCR)” and “no disease detected but incomplete evaluation to establish CR”.
Question 76: Specify disease status if not in complete remission
For recipients Not in complete remission, indicate whether clinical evidence of disease persisted on disease-specific assessments within the reporting period. If all assessments have shown resolution of disease, but not all assessments required to report complete remission have been completed, indicate No disease detected but incomplete evaluation to establish CR. This option is also appropriate for scenarios in which the recipient has not previously achieved a post-HCT CR but does not have any disease assessments performed within the reporting period. If disease persists by any radiological or clinical / hematological assessment indicate Disease detected. Persistence of abnormalities by molecular, cytogenetic, or flow cytometry assessments does not constitute ‘disease detected’ and should not be reported as disease detected for this question.
Example 2: A recipient with multiple myeloma goes to transplant in VGPR, without a bone marrow showing < 5% plasma cells completed prior to transplant. Post-transplant serum and urine electrophoreses and immunofixations are negative. However, no bone marrow biopsy is performed within the 100-day reporting period. In this case, “not in complete remission” should be selected for the best response, and “no disease detected by incomplete evaluation to establish CR” for specifying the disease status if not in complete remission data field.
Example 3: A recipient with AML goes to transplant in primary induction failure. Post-transplant, they recover their counts, but had circulating blasts noted on differential. They expire due to persistent disease with their last CBC performed on their date of death showing circulating blasts. In this case, “not in complete remission” should be selected for the best response to HCT, and “disease detected” for specifying the disease status if not in complete remission data field.
Example 4: Similar to example 2, a recipient with AML goes to transplant in primary induction failure. They expire on D+11 due to infection, and had not engrafted as of that date. Their last CBC showed a WBC of 0.5 × 109/L with no blasts detected on their differential. A bone marrow biopsy was not performed between transplant and the date of death. In this case, “not in complete remission” should be selected for the best response to HCT, and “no disease detected by incomplete evaluation to establish CR” for specifying the disease status if not in complete remission data field.
Question 77: Was the date of best response previously reported?
Indicate whether complete remission was reported in a previously reporting period.This question does not apply if the best response is Not in complete remission.
Question 78: Date assessed
Report the date complete remission was achieved. This date should fall after transplant but before or on the date of contact for the current reporting period. This should reflect the date of specimen collection or imaging for the latest assessment required to fulfill complete remission criteria for the recipient’s transplant disease.
Disease Assessment at Time of Best Response
The disease assessment questions (i.e., molecular, flow cytometry, cytogenetic, radiologic and clinical / hematologic assessments) refer to disease assessments performed at the time of best response (Date assessed). The following guidelines should be used to determine whether testing was performed at the time of best response:
If the recipient’s best response is Not in Complete Remission, report the latest assessment performed during the reporting period. If the recipient never achieved a CR and started treatment for progressive disease (excluding treatment for minimal residual disease), report the most recent assessments prior to progression. Review example E and F below.
If the recipient’ best response is Complete Remission, report testing performed closest to the date of best response (Date assessed) and within the time windows in the Disease Assessment Time Windows table.
Disease Assessment Time Windows
| Follow-Up Form | Approximate Range |
|---|---|
| 100 Day | +/- 15 days of date of best response (Date assessed) |
| 6 Month | +/- 15 days of date of best response (Date assessed) |
| Annual | +/- 30 days of date of best response (Date assessed) |
Disease Assessment Reporting Scenarios:
A. A recipient receives a transplant on 1/1/2015 for multiple myeloma in partial remission. Prior to HCT, FISH testing detects an IGH rearrangement associated with the recipient’s primary disease. During the 100 day reporting period, the recipient achieves a very good partial remission. FISH testing is only performed on 2/1/2015 is positive for the previously detected IGH rearrangement. The 100 day date of contact is 4/15/2015. In this case, the center would report the recipient was “Not in Complete Remission” on the 100 Day Post-TED Form. The center would report FISH testing was performed on 2/1/2015. When the best response is “Not in Complete Remission” report the most recent testing performed during the reporting period (assuming treatment was not started for relapsed, progressive, or persistent disease during the reporting period – see Scenario B).
B. A recipient receives a transplant on 1/1/2015 for multiple myeloma in partial remission. Prior to HCT, FISH testing detects an IGH rearrangement associated with the recipient’s primary disease. During the 100 day reporting period, the recipient has disease progression and starts treatment on 3/1/2015. FISH testing is performed on 2/1/2105 and 3/15/2015. Both tests are positive for the previously detected IGH rearrangement. The 100 day date of contact is 4/15/2015. In this case, the center would report the recipient was “Not in Complete Remission” on the 100 Day Post-TED Form. The center would report FISH testing was performed on 2/1/2015. When the best response is “Not in Complete Remission” report the most recent testing performed during the reporting period and prior to treatment for relapsed, progressive or persistent disease.
Note: For all subsequent reporting periods, the center would report “Not Evaluated” for the best response to HCT and skip to the Post-HCT Therapy section of the form. If treatment was started in a prior reporting period, the center is not able to report and assessments performed during the reporting period and prior to treatment
C. A recipient receives a transplant on 1/1/2015 for AML in primary induction failure. Prior to HCT, molecular testing confirms the recipient’s disease is FLT3 positive. On 2/1/2015, the recipient achieves a hematologic remission, but FLT3 is not tested at that time. Later, on 2/10/2015, molecular testing is performed and confirms the recipient is FLT3 negative. In this case, the center would report the recipient achieved a CR on 2/1/2015 on the 100 Day Post-TED Form. The center would report molecular testing was performed at the time of best response as testing was done within 15 days of 2/1/2015.
D. A recipient receives a transplant on 1/1/2015 for AML in primary induction failure. Prior to HCT, molecular testing confirms the recipient’s disease is FLT3 positive. On 2/1/2015, the recipient achieves a hematologic remission, but FLT3 is not tested at that time. Later, on 3/1/2015, molecular testing is performed and confirms the recipient is FLT3 negative. In this case, the center would report the recipient achieved a CR on 2/1/2015 on the 100 Day Post-TED Form. The center would report no molecular testing was performed at the time of best response as testing was not done within 15 days of 2/1/2015.
E. A recipient receives a transplant on 1/1/2015 for NHL in stable disease. During the 100 Day reporting period, a PET / CT was performed on Day 60, confirming stable disease but then on Day 95, another PET / CT was performed and showed progression. As a result, therapy for progression began on Day 100. The best response to HCT for the Day 100 reporting period would be reported as “Not in complete remission – disease detected” and report “Yes,” radiologic assessments were performed with the Day 60 PET / CT as this is the most recent scan prior to progression.
F. A recipient receives a transplant on 1/1/2020 for IgA Kappa Multiple Myeloma in stable disease. During the 100 Day reporting period, the first set of myeloma labs on Day 29, 1/30/2020, show progressive disease. Myeloma labs repeated on Day 60 and Day 100 also showed disease progression. As a result, therapy is planned to be given, starting in the 6-month reporting period, on Day 110. The best response to HCT for the Day 100 reporting period would be reported as “Not in complete remission – disease detected” and report “Yes,” clinical / hematologic assessments were performed with the Day 100 myeloma labs, as this is the most recent testing in the reporting period. In cases where the first assessment post-HCT shows progression, report the last assessment prior to the start of treatment. If treatment doesn’t start until the next reporting period, report the last assessment in the current reporting period.
Molecular
Question 79: Was the disease status assessed by molecular testing (e.g. PCR)?
Molecular assessment involves determining whether a molecular marker for the disease exists in the blood or bone marrow. Molecular assessment is the most sensitive method of detection and can indicate known genetic abnormalities associated with the disease for which the HCT was performed. Molecular assessments include polymerase chain reaction (PCR) amplification to detect single specific disease markers; however, molecular methods are evolving and now include Sanger sequencing and next generation sequencing (e.g., Illumina, Roche 454, Proton / PGM, SOLiD). Molecular marker results identified by FISH or chromosomal microarray assessments should not be reported as molecular testing.
Report Not applicable if molecular studies were never performed or have never shown abnormalities associated with the recipient’s primary transplant disease.
Once an assessment is positive for disease, Not applicable will never be an appropriate response.
Report No if molecular studies were positive for disease in the past but not performed during the current reporting period.
If the recipient’s best response is Not in Complete Remission, report Yes if assessments were performed, prior to any progression or treatment for persistent / progressive disease, excluding treatment for minimal residual disease). Report No if assessments were not performed, prior to any progression or treatment for persistent disease (excluding treatment for minimal residual disease).
If the recipient’s best response is Complete Remission, report Yes if disease assessments were performed within the time windows listed in the Disease Assessments Time Windows table above. If testing was not performed within the applicable time window, report No.
Question 80: Date assessed
If the best response is Complete remission, report the date of testing performed nearest the date of best response (within the time windows listed in the Disease Assessments Time Windows table above) and prior to relapse or progression, if applicable.
If the best response is Not in complete remission, report the date of the most recent testing performed during the reporting period and prior to progression or treatment for persistent disease, if applicable.
Report the date of specimen collection for molecular disease assessment. If exact date is not known, refer to General Instructions, General Guidelines for Completing Forms for information about reporting partial or unknown dates.
Question 81: Was disease detected?
Report whether the recipient’s primary disease was detected by molecular testing on the date reported. This question may be reported as Yes even when Not in complete remission – no disease detected but incomplete evaluation to establish CR is reported as the best response. In order to be considered positive for disease, the assay must detect a number of copies of the molecular marker exceeding the threshold for sensitivity of the assay, for a quantitative study. However, do note that presence of only a single marker among numerous tested is sufficient to indicate disease detected.
Flow Cytometry
Question 82: Was the disease status assessed via flow cytometry?
Flow cytometry is a technique that can be performed on blood, bone marrow, or tissue preparations where cell surface markers can be quantified on cellular material. This allows for the detection of abnormal cell populations for some diseases.
Report Not applicable if flow cytometry was never performed or have never shown abnormalities associated with the recipient’s primary transplant disease.
Once an assessment is positive for disease, Not applicable will never be an appropriate response.
Report No if flow cytometry studies were positive for disease in the past but not performed during the current reporting period.
If the recipient’s best response is Not in Complete Remission, report Yes if assessments were performed, prior to any progression or treatment for persistent / progressive disease, excluding treatment for minimal residual disease). Report No if assessments were not performed, prior to any progression or treatment for persistent disease (excluding treatment for minimal residual disease).
If the recipient’s best response is Complete Remission, report Yes if disease assessments were performed within the time windows listed in the Disease Assessments Time Windows table above. If testing was not performed within the applicable time window, report No.
Question83: Date assessed
If the best response is Complete remission, report the date of testing performed nearest the date of best response (within the time windows listed in the Disease Assessments Time Windows table above) and prior to relapse or progression, if applicable.
If the best response is Not in complete remission, report the date of the most recent testing performed during the reporting period and prior to progression or treatment for persistent disease, if applicable.
Report the date of specimen collection for flow cytometry assessment. If exact date is not known, refer to General Instructions, General Guidelines for Completing Forms, for information about reporting partial or unknown dates.
Question 84: Was disease detected?
Report whether the recipient’s primary disease was detected by flow cytometry on the date reported in question 93. Report Yes if an abnormal cell population associated with the recipient’s primary transplant disease was detected regardless of the sensitivity of the flow cytometry panel performed; this means an abnormal cell population detected by MRD flow cytometry would be reported in the same way as an abnormal cell population detected by a standard flow cytometry assay. This question may be reported Yes even when Not in complete remission – no disease detected but incomplete evaluation to establish CR is reported as the best response.
Cytogenetic Testing (Karyotyping or FISH)
Question 85: Was the disease status assessed by cytogenetic testing (karyotyping or FISH)?
Cytogenetic studies involve the study of chromosomes, typically through one of two methods: karyotyping or fluorescence in situ hybridization (FISH). Blood, bone marrow, or tissue preparations may be tested by either of these two methods. Karyotyping is both less sensitive and less specific than FISH testing; FISH studies identify only abnormalities detectable by the employed probe set, and cannot provide information about the presence or absence of chromosomal abnormalities or markers outside the specific probe set utilized.
Report Not applicable if cytogenetic studies were never performed or have never shown abnormalities associated with the recipient’s primary transplant disease.
Once an assessment is positive for disease, Not applicable will never be an appropriate response.
Report No if cytogenetic studies were positive for disease in the past but not performed during the current reporting period.
If the recipient’s best response is Not in Complete Remission, report Yes if assessments were performed, prior to any progression or treatment for persistent / progressive disease, excluding treatment for minimal residual disease). Report No if assessments were not performed, prior to progression or treatment for persistent disease (excluding treatment for minimal residual disease).
If the recipient’s best response is Complete Remission, report Yes if disease assessments were performed within the time windows listed in the Disease Assessments Time Windows table above. If testing was not performed within the applicable time window, report No.
Question 86: Was the disease status assessed via FISH?
FISH XX/XY probe sets are not considered relevant to disease assessment and should not be reported in the disease assessment section.
Chromosomal microarrays / chromosomal genomic arrays should be reported as FISH assessments.
Report Not applicable if FISH studies were never performed or have never shown abnormalities associated with the recipient’s primary transplant disease.
Once an assessment is positive for disease, Not applicable will never be an appropriate response.
Report No if FISH studies were positive for disease in the past but not performed during the current reporting period.
If the recipient’s best response is Not in Complete Remission, report Yes if assessments were performed, prior to any progression or treatment for persistent / progressive disease, excluding treatment for minimal residual disease). Report No if assessments were not performed, prior to any progression or treatment for persistent disease (excluding treatment for minimal residual disease).
If the recipient’s best response is Complete Remission, report Yes if disease assessments were performed within the time windows listed in the Disease Assessments Time Windows table above. If testing was not performed within the applicable time window, report No.
Question 87: Date assessed
If the best response is Complete remission, report the date of testing performed nearest the date of best response (within the time windows listed in the Disease Assessments Time Windows table above) and prior to relapse or progression, if applicable.
If the best response is Not in complete remission, report the date of the most recent testing performed during the reporting period and prior to progression or treatment for progressive disease, if applicable.
Report the date of specimen collection for FISH assessment. If exact date is not known, refer to General Instructions, General Guidelines for Completing Forms, for information about reporting partial or unknown dates.
Question 88: Was disease detected?
Report whether the recipient’s primary disease was detected by FISH testing on the date reported. This question maybe reported as Yes even when Not in complete remission – no disease detected but incomplete evaluation to establish CR is reported as the best response.
Question 89: Was the disease status assessed via karyotyping?
Report Not applicable if karyotyping was never performed or have never shown abnormalities associated with the recipient’s primary transplant disease.
Once an assessment is positive for disease, Not applicable will never be an appropriate response.
Report No if karyotyping studies were positive for disease in the past but not performed during the current reporting period.
If the recipient’s best response is Not in Complete Remission, report Yes if assessments were performed, prior to any relapse, progression or treatment for persistent disease, (excluding treatment for minimal residual disease). Report No if assessments were not performed, prior to any relapse, progression, or treatment for persistent disease (excluding treatment for minimal residual disease).
If the recipient’ best response is Complete Remission, report Yes if disease assessments were performed within the time windows in the Disease Assessments Time Windows table above. If testing was not performed within the applicable time window, report No.
Question 90: Date assessed
If the best response is Complete remission, report the date of testing performed nearest the date of best response (within the time windows listed in the Disease Assessments Time Windows table above) and prior to relapse or progression, if applicable.
If the best response is Not in complete remission, report the date of the most recent testing performed during the reporting period and prior to progression or treatment for persistent disease, if applicable.
Report the date of specimen collection for karyotyping. If exact date is not known, refer to General Instructions, General Guidelines for Completing Forms, for information about reporting partial or unknown dates.
Question 91: Was disease detected?
Report whether the recipient’s primary disease was detected by karyotyping on the date reported. Do not include clinically insignificant polymorphism, or chromosomal abnormalities of no known significance, as disease detected; this includes anomalies such as age-dependent loss of the chromosome Y. This question may be reported as Yes even when Not in complete remission – no disease detected but incomplete evaluation to establish CR is reported as the best response.
Radiologic
Question 92: Was the disease status assessed by radiological assessment (e.g. PET, MRI, CT)
Radiologic assessments are imaging techniques used to assess disease response to transplant, typically for lymphomas or solid tumors, though valuable in some less common presentations of disease, such as leukemia cutis. Imaging techniques used to evaluate disease response typically include PET, CT, or MIBG, but may include x-ray, skeletal survey, or ultrasound in some cases.
Report Not applicable if radiological assessments were never performed or have never shown abnormalities associated with the recipient’s primary transplant disease.
Once an assessment is positive for disease, Not applicable will never be an appropriate response.
Report No if radiological assessments were positive for disease in the past but not performed during the current reporting period.
If the recipient’s best response is Not in Complete Remission, report Yes if assessments were performed, prior to any progression or treatment for persistent / progressive disease, excluding treatment for minimal residual disease). Report No if assessments were not performed, prior to any progression or treatment for persistent disease (excluding treatment for minimal residual disease).
If the recipient’s best response is Complete Remission, report Yes if disease assessments were performed within the time windows listed in the Disease Assessments Time Windows table above. If testing was not performed within the applicable time window, report No.
Question 93: Date assessed
If the best response is Complete remission, report the date of the assessment performed nearest the date of best response (within the time windows listed in the Disease Assessments Time Windows table above) and prior to relapse or progression, if applicable.
This date may match the date CR was achieved reported in Date assessed for the best response for recipients with lymphomas, solid tumors, or other diseases with imaging criteria for reporting CR.
If the best response is Not in complete remission – no disease detected but incomplete evaluation to establish CR, report the last assessment performed in the reporting period and prior to progression or treatment for persistent / progressive disease, if applicable.
If the best response is Not in complete remission – disease detected, report the most recent radiological testing performed in the reporting period that detects disease and prior to progression or treatment for persistent disease, if applicable. If disease was not detected by this method report the date of the most recent radiological testing performed and prior to progression or treatment for persistent disease, if applicable.
If exact date is not known, refer to General Instructions, General Guidelines for Completing Forms, for information about reporting partial or unknown dates.
Question 94: Was disease detected?
Report whether the recipient’s primary disease was detected by radiologic assessment on the reported date.
Clinical / Hematologic
Question 95: Was the disease status assessed by clinical / hematologic assessment?
Clinical / hematologic disease assessments are the least sensitive method of disease detection. Examples include circulating blasts in the bloodstream for AML, and enlargement of a malignant mass for lymphoma or a solid tumor on physical examination. Every recipient who has an evaluation by a physician has a “clinical” assessment. Do not include radiologic or imaging assessments when answering this question.
If the recipient’s best response is Not in Complete Remission, report Yes If assessments were performed prior to progression or treatment for persistent disease, (excluding treatment for minimal residual disease). Report No If assessments were not performed prior to progression or treatment for persistent disease, (excluding treatment for minimal residual disease).
If the recipient’ best response is Complete Remission, report Yes if disease assessments were performed within the time windows in the Disease Assessment Time Windows table above. If testing was not performed within the applicable time window, report No.
Question 96: Date assessed
If the best response is Complete remission, report the date of the assessment performed nearest the date of best response (within the time windows listed in the Disease Assessments Time Windows table above) and prior to relapse or progression, if applicable. This will likely match the date CR reported in Date assessed for the best response, since complete remission criteria generally require clinical or hematologic assessment to confirm.
If the best response is Not in complete remission – no disease detected but incomplete evaluation to establish CR, report the last assessment performed in the reporting period and prior to progression or treatment for persistent disease, if applicable.
If the best response is Not in complete remission – disease detected, report the most recent assessment performed in the reporting period that detects disease and prior to progression or treatment for persistent disease, if applicable. If disease was not detected by this method report the date of the most recent assessment performed and prior to progression or treatment for persistent disease, if applicable.
If exact date is not known, refer to General Instructions, General Guidelines for Completing Forms, for information about reporting partial or unknown dates.
Question 97: Was disease detected?
Report whether clinical / hematologic abnormalities associated with the primary disease were detected. In general, if the clinical / hematologic assessment date is the same as the reported Date assessed for the best response, for recipients achieving complete remission in the reporting period, the answer to this question should be No.
Section Updates:
| Question Number | Date of Change | Add/Remove/Modify | Description | Reasoning (If applicable) |
|---|---|---|---|---|
| . | . | . | . | . |
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