Perspectives on Drug Development for the Treatment of Chronic Myeloid Leukemia in Pregnant Patients and Patients Who Are Breastfeeding (2024)

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Volume 30, Issue 17

1 September 2024

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CCR Perspectives in Regulatory Science and Policy| September 03 2024

Jorge E. Cortes

;

Jorge E. Cortes *

1

Georgia Cancer Center at Augusta University, Augusta, Georgia.

*Corresponding Author: Jorge E. Cortes, Georgia Cancer Center, 1410 Laney Walker Road, CN2222, Augusta, GA 30912. E-mail: jorge.cortes@augusta.edu

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Elisabetta Abruzzese

;

Elisabetta Abruzzese

2

Hematology, S. Eugenio Hospital, Tor Vergata University, Rome, Italy.

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Elyce H. Cardonick

;

Elyce H. Cardonick

3

Cooper Medical School of Rowan University, Camden, New Jersey.

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Sonia Hernández-Díaz

;

Sonia Hernández-Díaz

4

Harvard T.H. Chan School of Public Health, Boston, Massachusetts.

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Jamie Gutierrez

;

Jamie Gutierrez

5

Patient advocate, Castle Pines, Colorado.

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Mary S. Sardegna

;

Mary S. Sardegna

6

Patient advocate, Laguna Hills, California.

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Erica Torres-Chavez

;

Erica Torres-Chavez

7

Patient advocate, Riverside, California.

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Miriam Dinatale

;

Miriam Dinatale

8

FDA, Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, Silver Spring, Maryland.

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Catherine C. Lerro

;

Catherine C. Lerro

9

FDA, Oncology Center of Excellence, Silver Spring, Maryland.

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Brenda J. Gehrke

;

Brenda J. Gehrke

10

FDA, Center for Drug Evaluation and Research, Office of Oncologic Diseases, Silver Spring, Maryland.

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Stacy S. Shord

;

Stacy S. Shord

11

FDA, Center for Drug Evaluation and Research, Office of Clinical Pharmacology, Silver Spring, Maryland.

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R. Angelo De Claro

;

R. Angelo De Claro

10

FDA, Center for Drug Evaluation and Research, Office of Oncologic Diseases, Silver Spring, Maryland.

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Marc R. Theoret

;

Marc R. Theoret

9

FDA, Oncology Center of Excellence, Silver Spring, Maryland.

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Peter J. DeMaria

;

Peter J. DeMaria

8

FDA, Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, Silver Spring, Maryland.

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Kelly J. Norsworthy

Kelly J. Norsworthy

10

FDA, Center for Drug Evaluation and Research, Office of Oncologic Diseases, Silver Spring, Maryland.

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Author & Article Information

*Corresponding Author: Jorge E. Cortes, Georgia Cancer Center, 1410 Laney Walker Road, CN2222, Augusta, GA 30912. E-mail: jorge.cortes@augusta.edu

Clin Cancer Res 2024;30:3658–66

Received: March 19 2024

Revision Received: May 17 2024

Accepted: July 03 2024

Online ISSN: 1557-3265

Print ISSN: 1078-0432

Funding

Funding Group:

  • Award Group:

    • Funder(s):

      U.S. Food and Drug Administration (FDA)

    • Principal Award Recipient(s):

      C.C.

      Lerro

      ,

      B.J.

      Gehrke

      ,

      S.S.

      Shord

      ,

      R.

      Angelo de Claro

      ,

      M.R.

      Theoret

      ,

      M.

      Dinatale

      ,

      P.J.

      DeMaria

      ,

      K.J.

      Norsworthy

©2024 American Association for Cancer Research

2024

American Association for Cancer Research

Clin Cancer Res (2024) 30 (17): 3658–3666.

Article history

Received:

March 19 2024

Revision Received:

May 17 2024

Accepted:

July 03 2024

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    • Version of Record September 3 2024
    • Proof July 23 2024
    • Accepted Manuscript July 5 2024

Citation

Jorge E. Cortes, Elisabetta Abruzzese, Elyce H. Cardonick, Sonia Hernández-Díaz, Jamie Gutierrez, Mary S. Sardegna, Erica Torres-Chavez, Miriam Dinatale, Catherine C. Lerro, Brenda J. Gehrke, Stacy S. Shord, R. Angelo De Claro, Marc R. Theoret, Peter J. DeMaria, Kelly J. Norsworthy; Perspectives on Drug Development for the Treatment of Chronic Myeloid Leukemia in Pregnant Patients and Patients Who Are Breastfeeding. Clin Cancer Res 1 September 2024; 30 (17): 3658–3666. https://doi.org/10.1158/1078-0432.CCR-24-0826

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Abstract

Tyrosine kinase inhibitors (TKI) have improved the outcome and life expectancy of patients with chronic myeloid leukemia (CML). Patients are diagnosed with CML at younger ages, and patients treated for CML may become pregnant or choose to breastfeed. The information available to date on the safety of TKIs during pregnancy and lactation and the optimal management of these patients is largely anecdotal, based on personal or small-group experience, and heterogeneous. A panel of interested parties was convened by U.S. Food and Drug Administration to analyze the current data and discuss possible solutions. Possible solutions include prospective data collection, in clinical trials and in routine clinical practice, a more uniform and specific data collection, and greater coordination among involved entities. As patients with cancer are living longer, frequently receiving therapies for extended periods of time (or for life), data on appropriate management of patients through different reproductive phases of life are needed. It is thus time to change our approach for how to study treatment of cancer (including CML) during pregnancy or breastfeeding to develop evidence-based guidelines for safe and effective patient care.

©2024 American Association for Cancer Research

2024

American Association for Cancer Research

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Perspectives on Drug Development for the Treatment of Chronic Myeloid Leukemia in Pregnant Patients and Patients Who Are Breastfeeding (2024)

FAQs

How is chronic myeloid leukemia treated in pregnancy? ›

Treatment options considered safe during pregnancy include leukapheresis and IFN. Treatment with IFN-α controls the high cell mass in the majority of newly diagnosed patients with CML. However, the degree of response ranges from no 'hematologic' response to complete suppression of the leukemic clone.

What is the drug of choice for chronic myeloid leukemia? ›

The most common treatment is a type of targeted cancer drug called a tyrosine kinase inhibitor (TKI). There are several different TKIs. The most common drug for CML is imatinib (Glivec). But your doctor might suggest a different TKI such as dasatanib (Sprycel) or nilotinib (Tasigna).

What are the pregnancy outcomes among patients with chronic myeloid leukemia treated with dasatinib? ›

Twenty of 46 pregnancies (43%) resulted in live births, with delivery of a normal infant at term in 15 (33%) and abnormal pregnancies in 5 (11%). Pregnancies were terminated in 26 of 46 cases (57%): 18 elective and 8 spontaneous abortions.

What are the treatment recommendations for chronic myeloid leukemia? ›

Treatment options for chronic-phase chronic myeloid leukemia (CML) include the following:
  • Targeted therapy with tyrosine kinase inhibitors (TKIs).
  • Allogeneic bone marrow transplant (BMT) or stem cell transplant (SCT).
Jul 15, 2024

How is AML treated in pregnancy? ›

Chemotherapy is the main treatment for AML. Doctors consider chemotherapy generally safe for a fetus during the second and third trimesters of pregnancy. However, in the first trimester, chemotherapy can be harmful to a fetus and may cause : fetal death.

Can you be treated for leukemia while pregnant? ›

Pregnant women with leukaemia need to be managed by a multidisciplinary team that includes haematologists, obstetricians, neonatologists, anaesthetists and pharmacists in order to manage all the treatments required. Treatment during the first trimester can be associated with congenital anomalies and miscarriage.

What is the first-line drug used to treat chronic myeloid leukaemia? ›

First-line treatment

If you're diagnosed in the chronic phase, your first treatment will usually be a regular standard dose of one of three types of TKI: imatinib, dasatinib or nilotinib.

What is the first choice drug for CML? ›

The most common treatment for chronic myeloid leukaemia (CML) is a type of targeted cancer drug called a tyrosine kinase inhibitor (TKI). Common TKIs for CML include imatinib, bosutinib, dasatinib and nilotinib.

What is the new drug for chronic leukemia? ›

The FDA granted accelerated approval in December 2023 to a new kind of BTK inhibitor, pirtobrutinib (Jaypirca), for adults with CLL or small lymphocytic lymphoma (SLL) who have had at least two prior lines of therapy.

How common is CML in pregnancy? ›

A diagnosis of CML during pregnancy is not an uncommon occurrence; approximately 10% of pregnancy-related leukemias are CML, and 20% of pregnancy-related CML is in patients diagnosed during pregnancy.

Is imatinib safe in pregnancy? ›

Around 200 women taking imatinib in early pregnancy have been studied. While most of their babies were healthy, a handful had a similar pattern of birth defects. This suggests that in rare cases, imatinib might affect the unborn baby's development.

What is the new treatment for chronic myeloid leukemia? ›

However, a new drug, asciminib, has shown promising results as a safer and more effective treatment for patients with newly diagnosed CML. The phase 3 ASC4FIRST trial included 405 patients recently diagnosed with chronic phase CML.

Which is the best drug for CML? ›

The first-line treatment for CML is tyrosine kinase inhibitors (TKIs), a type of targeted therapy drug. TKIs include : imatinib (Gleevec) dasatinib (Sprycel)

What is the gold standard treatment for chronic myeloid leukaemia? ›

The standard treatment of choice for chronic phase CML is a TKI: either the first-generation TKI imatinib, which is a specific small-molecule inhibitor of BCR-ABL in all phases of CML, or a second-generation TKI—nilotinib (Tasigna), dasatinib (Sprycel), or bosutinib (Bosulif).

What is the initial treatment for chronic myeloid leukemia? ›

Treatment of chronic phase chronic myelogenous leukemia may include the following: Targeted therapy with a tyrosine kinase inhibitor (imatinib mesylate, nilotinib, dasatinib, bosutinib). High-dose chemotherapy with donor stem cell transplant. Chemotherapy.

Can you have a baby if you have CML? ›

Growing numbers of CML patients of childbearing age are living in stable remissions and are considering having children while being treated for CML. There is no risk that parents will pass the Ph chromosome onto their children. Generally, there are no concerns for men on TKIs associated with having children.

What is the best treatment for myeloid leukemia? ›

The main treatment for most types of AML is chemotherapy, sometimes along with a targeted therapy drug. This might be followed by a stem cell transplant. Other drugs (besides standard chemotherapy drugs) may be used to treat people with acute promyelocytic leukemia (APL).

What is the first line treatment for chronic myeloid leukemia? ›

First-line treatment

If you're diagnosed in the chronic phase, your first treatment will usually be a regular standard dose of one of three types of TKI: imatinib, dasatinib or nilotinib.

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